Category Archives: Stem Cell Medicine

Gamida Cell Announces Results from Phase 1 Study of GDA-201 and New Mechanism of Action Data at ASH 2019 Annual Meeting – Business Wire

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, today announced updated results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM), at the 61st Annual Meeting of the American Society of Hematology (ASH), which is being held December 710 in Orlando, FL. Data from 22 patients in the ongoing study showed GDA-201 in combination with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity in heavily pre-treated patients, including five complete responses observed among nine patients with NHL. Gamida Cell plans to initiate a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with NHL in 2020.

NK cells are increasingly recognized as a potential breakthrough approach in immunotherapy, and the data reported today provide early evidence that GDA-201 has the potential to be an important new treatment option, said Veronica Bachanova, M.D., Ph.D., Associate Professor of Medicine in the Division of Hematology, Oncology and Transplantation at the University of Minnesota and principal investigator of the study through the Masonic Cancer Center. Given the population of heavily pre-treated patients with advanced disease, its particularly encouraging to witness multiple complete responses. I look forward to the continued development of this investigational therapy.

New research was also presented today on the mechanism of action of Gamida Cells NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes observed in the Phase 1/2 clinical study of omidubicel, the companys advanced cell therapy currently in Phase 3 clinical development as a potential life-saving treatment option for patients in need of an allogeneic bone marrow transplant.

These mechanism of action data reinforce the transformative potential of our NAM therapeutic platform, which can be used to expand multiple cell types. Specifically for omidubicel, this research suggests that NAM modulates certain gene expression pathways that, collectively, mimic the hypoxic environment of the bone marrow to help preserve stem cell function and long-term engraftment ability, said Tracey Lodie, Ph.D., chief scientific officer of Gamida Cell. We expect to build on our findings by characterizing the metabolites produced when we expand stem cells to make omidubicel, and we are also beginning to conduct similar mechanism of action studies with GDA-201.

GDA-201 Phase 1 Clinical Data Presented at ASH

The oral presentation, Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM) (Abstract #777), described data from the Phase 1 clinical study of GDA-201 in heavily pre-treated patients with advanced NHL and MM. Twenty-two patients were enrolled in the study, including nine patients with NHL and 13 patients with MM. Of these 22 patients, all were evaluable for safety and 21 were evaluable for response (NHL = 9; MM = 12).

In the study, cell therapy using GDA-201 with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity. Of the nine patients with NHL, five achieved a complete response and one achieved a partial response. Among the patients with MM, one patient achieved a complete response, and five patients achieved stable disease.

GDA-201 was generally well tolerated, with no graft vs. host disease (GvHD), no tumor lysis syndrome, no neurotoxicity and no marrow aplasia observed. No dose limiting toxicities were observed. Hypertension and hematologic events were the most common Grade 3/4 adverse events observed. Most non-hematologic toxicities were attributed to cyclophosphamide/fludarabine, which was used as a pre-conditioning treatment.

NAM Therapeutic Platform Mechanism of Action Data Presented at ASH

The poster presentation, Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential (Abstract #3718), included transcriptome, transcription factor, and pathway analysis to elucidate the pathways leading to the preservation of engraftment after ex vivo expansion of CD34+ hematopoietic stem cells derived from umbilical cord blood (the starting point for omidubicel) compared to CD34+ cells grown in the absence of NAM.

Analyses showed that the presence of NAM reduced the expression of genes involved in the production of reactive oxygen and nitrogen species, suggesting that cell stress was minimized during expansion. In addition, NAM also decreased growth factor pathways responsible for activation and differentiation of hematopoietic stem cells, suggesting NAM expanded cells while keeping them in an undifferentiated state. The presence of NAM also led to a decrease in the expression of genes responsible for matrix-metallo proteinase secretion, simulating the microenvironment of the bone marrow. Additionally, NAM led to an increased expression of telomerase genes, which is believed to enable cells to remain in a more quiescent, stem-like state. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes that were observed in the Phase 1/2 clinical study of omidubicel.

About GDA-201GDA-201 (formerly known as NAM-NK) is being developed as an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. NK cells have the ability to kill tumor cells, representing a novel immunotherapeutic approach to cancer treatment. GDA-201 is designed to address key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1 For more information on the clinical study of GDA-201, please visit http://www.clinicaltrials.gov.

About OmidubicelOmidubicel (formerly known as NiCord), the companys lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.2 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Latin America, Europe and Asia.3 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.4 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

GDA-201 and omidubicel are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic PlatformGamida Cells proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types including stem cells and natural killer (NK) cells with appropriate growth factors to maintain the cells original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

About Gamida CellGamida Cell is an advanced cell therapy company committed to finding cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com.

Cautionary Note Regarding Forward Looking StatementsThis press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the initiation of new clinical trials and the continuation of the Companys clinical development program, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope and progress of Gamida Cells clinical trials and other clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of Gamida Cells public filing on Form 20-F, filed with the SEC on February 25, 2019, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1ClinicalTrials.gov identifier NCT03019666.2 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.3 ClinicalTrials.gov identifier NCT027302994 ClinicalTrials.gov identifier NCT03173937

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Gamida Cell Announces Results from Phase 1 Study of GDA-201 and New Mechanism of Action Data at ASH 2019 Annual Meeting - Business Wire

AgeX Therapeutics Issues Year-End Letter to Shareholders – BioSpace

The letter follows.

Dear AgeX Stockholders,

In this, our first year as a public company, we have built a foundation for a revolutionary company in the fields of cell therapy and tissue regeneration. To date, conventional pharmaceutical approaches to the chronic degenerative conditions associated with aging have provided little benefit, often only offering relief from the symptoms of disease, rather than targeting underlying disease processes. Our belief is that this is about to change through harnessing the power of new cellular and molecular technologies. We aim to lead this coming revolution with our pioneering technologies which could generate and deliver new cells to patients through our cell therapy focus, and which may reverse the age of cells already in the body through our iTR platform. We believe that our new technologies will lead to true cell regeneration and replacement to potentially cure degenerative diseases by targeting aged or damaged cells, tissues and organs.

Over the last year, we have worked hard to achieve certain goals to set the fundamental basis to create shareholder value going forward:

To optimize shareholder value, we have undertaken a strategic review of our business opportunities, and we have four key take-away messages for the coming year and beyond:

UniverCyte would potentially be game-changing for the whole cell therapy industry by allowing the transplantation of non-self, donor cells into all patients without the need for powerful immunosuppressant drugs, which are associated with serious side effects, including infections and cancers, as well as kidney and liver toxicity. The UniverCyte platform aims to utilize a proprietary, novel, modified form of the powerful immunomodulatory molecule HLA-G, which in nature seems to be a dominant player in protecting a baby from destruction by the mother's immune system during pregnancy, the only known physiological state of immune tolerance toward foreign tissue in humans.

On the other hand, our pluripotent stem cell-based PureStem platform could potentially overcome numerous industry barriers. PureStem cells would have eight potential advantages compared to other adult stem cell- or pluripotent stem cell-based therapies, including lower manufacturing costs, industrial scalability, off-the-shelf usage, high purity, non-tumorgenicity, young age (so they are not prone to the disadvantages associated with older cells), aptitude for permanent cell engraftment, and potential to manufacture any human cell type.

We have two in-house product candidates, both targeting highly prevalent diseases of old age, with a high unmet medical need, and which are for multi-billion-dollar markets. Our lead internal program going forward will be AgeX-BAT1, which is brown fat cells for the treatment of type II diabetes. The last year has seen significant investment in cell therapy product candidates for diabetes by investors and large biotech. Earlier this year, we published a paper, Clonal Derivation of White and Brown Adipocyte Progenitor Cell Lines from Human Pluripotent Stem Cells, in the peer-reviewed scientific journal Stem Cell Research & Therapy, which showed that our PureStem platform generated highly pure, identifiable and scalable brown adipose cells, expressing active adipokines. Our second internal program will be AgeX-VASC1, composed of vascular endothelial progenitor cells for tissue ischemia, such as peripheral vascular disease and potentially cardiac and CNS ischaemia. Once we have a UniverCyte-modified pluripotent stem cell cGMP master cell bank, we will re-derive universal versions of AgeX-BAT1 and AgeX-VASC1 and then work to establish proof-of-concept in animal models.

We care deeply about our mission and the needs of our stockholders. We appreciate your support and the dedication of our scientists and employees as we forge a new future for medicine. We invite you to join us for the Annual Meeting of Stockholders on Monday, December 30, 2019. For those of you who cannot attend in person, our corporate update from that meeting will be webcast for your convenience.

Sincerely,

Michael D. West, Ph.D.

Gregory Bailey, M.D.

Chief Executive Officer

Chairman of the Board

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

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AgeX Therapeutics Issues Year-End Letter to Shareholders - BioSpace

Jasper Therapeutics Announces Upcoming Data Presentation on Lead Program, JSP191, at 61st American Society of Hematology (ASH) Annual Meeting &…

JSP191, a humanized antibody targeting CD117, is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplantation. The Phase 1 clinical trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that had poor outcomes.

Life-threatening disorders such as SCID, and other conditions including autoimmune diseases and hematologic cancers, can be cured by hematopoietic cell transplantation, and those with certain genetic diseases can be cured with stem cell-directed gene therapies. However, the number of patients who can benefit from these approaches is limited because of the severe toxicity of the chemotherapy used for pre-transplant conditioning that is needed to allow room in the bone marrow for the stem cells to engraft, said Judith Shizuru, M.D., Ph.D., co-founder and member of the Board of Directors of Jasper Therapeutics. We are encouraged by the initial Phase 1 study results of JSP191 in these fragile patients with SCID and plan to expand clinical development of this antibody beyond patients with SCID. We expect to initiate clinical trials of JSP191 in 2020 to evaluate it as a conditioning agent in patients undergoing hematopoietic cell therapy for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia, and IND-enabling studies for sickle cell disease and autoimmune indications.

Details of the oral presentation follow:

Abstract Title: Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency (abstract #800) Session Name: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Innovative Approaches in Allogeneic Transplantation for Pediatric or Nonmalignant DisordersPresenter: Rajni Agarwal, M.D., Associate Professor of Pediatrics and Stem Cell Transplantation, the Stanford University School of MedicineTime: 3:00 p.m. ETLocation: W311EFGH, Level 3, Orange County Convention Center

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

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Jasper Therapeutics Announces Upcoming Data Presentation on Lead Program, JSP191, at 61st American Society of Hematology (ASH) Annual Meeting &...

The Uncertainty of Regulating 3D Organ Printing – The Regulatory Review

Policymakers around the world are determining how to apply existing regulations to 3D organ printing.

When Selena Gomez suffered from Lupus, her best friend Francia Raisa donated her kidney to her, saving Gomezs life. For decades, people just like Gomez have escaped death due to heroic organ donations, either from living or deceased donors.

Today, 3D printingalso referred to as 3DPmay revolutionize the practice of organ donation. Although 3D printing is currently used to make jewelry, food, and art, it may soon be used for medical solutions such as organ donations and bionic limbs.

As a result, policymakers around the world seek increased regulation of 3DP organs. Yet 3D bioprinting does not clearly fit into existing regulatory frameworks.

Since bioprinting generally falls within the regulatory domain of regenerative medicine, medical devices, and biologic drugs, regulators face the challenge of applying existing rules to this uncertain field. As of now, it is unclear whether policymakers can effectively regulate bioprinting under existing regulations, or if a new, specific regulatory process will be necessary.

In determining how to regulate 3D organ printing, policymakers must juggle many possible concerns. Since the technology is still developing, a lot of uncertainty remains about what the actual risks and ethical concerns are. For example, one ethical concern is that 3DP organs may be available to wealthy people only, while less affluent individuals will be blocked out from using these organs.

Another concern is safety. Since 3DP may require stem-cell technology, and the patients own cells may be used for replication, it is difficult to assess the safety risks. Stem-cell therapy cannot be tested on a large sample of healthy people, which limits effective clinical analysis. Also, 3DP biotechnology may open up new uses beyond 3DP organs, such as enhancement of human capacities for military use. Developers could use the technology to make military officers or even terrorists less vulnerable to injury in battle, but this would open up a whole new challenge for law enforcement and national security.

The U.S. Food and Drug Administration (FDA) focuses on the regulation of 3D printed organs. FDA so far has only released guidance on 3DP, and the recommendations do not cover bioprinting.

A significant concern in the United States is that 3D printed organs do not fit into any clear category of law. First, they are not organs because they are not born alive at any stage of development. Second, they are not drugs because drugs are used orally rather than through an invasive surgery, and drugs are primarily meant to relieve illness while donated organs may completely cure an illness.

Some policymakers in the United States propose regulating 3DP organs as a biological product, defined as a virus, therapeutic serum, toxinor analogous productapplicable to the prevention, treatment, or cure of a disease or condition of human beings.

Research company Biogelx suggests that biological products may be a promising category for printed organs. Within biological products, a 3DP organ is comparable to proteins because to print the organ, clinicians replicate healthy human cells, which include such proteins, says Biogelx. Although existing regulatory frameworks often compare 3DP organs to medical devices, Biogelx asserts that these organs should not be regulated as medical devices. Medical devices are not made of biological material and are often metal or plastic devices that help an individuals standard of life, but 3DP organs are different since they cause a chemical reaction in the body and have the purpose of wholly replacing an existing organ, says Biogelx.

International policymakers are also struggling to find a sufficient regulatory framework. In Canada, Health Canada released draft guidance last year to develop regulations for medical device manufacturers working towards bioprinting. Health Canada has several concerns about bioprinting, and it suggests that manufacturers looking for bioprinting licenses should be required to submit information regarding the use of additives in materials, the verification of the software for the bioprinting design, the method of sterilizing the machines, and the process of safe removal and reuse of bioprinting materials and residues.

Finally, Europes 3DP health technology is regulated by the European Medical Devices Directive, the Active Implantable Medical Devices Directive, and the Invitro Diagnostic Medical Devices Directive. The Medical Devices Directive categorizes bioprinting devices into several risk classes. Across the different classes, devices ranked as higher risk are subjected to third party assessment and more stringent requirements for clinical data. The highest risk class, implantable devices such as 3D organs, requires an independent design dossier review. A design dossier assesses risk, evaluates clinical data, and demonstrates the technologys compliance with regulations and requirements.

Although 3D printing of organs is right around the corner, policymakers around the world lack the information necessary to make regulatory decisions in this space. Different countries have different approaches, but many of the leading nations in 3DP share similar concerns. With more information, regulators will have to decide if existing regulatory frameworks can adequately address the safety concerns of 3D printed organs.

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The Uncertainty of Regulating 3D Organ Printing - The Regulatory Review

Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options – Business Wire

ORLANDO, Fla.--(BUSINESS WIRE)--Amnion of Florida, a leading provider of alternative medicine utilizing cryopreserved placental cell allograft and advanced bioactive facial rejuvenation, is pleased to announce their vendor choice to round out regenerative anti-aging therapies.

Amnion announces a partnership with Merakris Therapeutics, LLC to advance the development of Merakris topical bioactive anti-aging hydrogel technology. Christopher Broderick, President, and Founder of Merakris Therapeutics stated, dedication to science-based outcomes is our primary focus, thus were delighted to be selected based upon our scientific approach to youth maintenance and rejuvenation technologies.

Amnion is focused on attracting women and men seeking affordable non-surgical options for youth maintenance via cell activated procedures, hair restoration, joint repair, dermal rejuvenation, and anti-aging treatments.

Our team of experienced medical professionals and aestheticians at Amnion are excited to utilize the Merakris Therapeutics product suite, including medical-grade, sterile filtered amniotic fluid serums and hydrogels at our newest Spa in Sanford, FL, said Eusebio Coterillo, President of Amnion.

In a constantly changing field, Amnion of Florida, under the guidance of the on-site medical staff, provides the highest level of quality products and procedures in cosmetic medicine. They offer cutting edge treatments that are proven by research, the use FDA cleared or registered products, and are widely published and peer endorsed.

More about Amnion of Florida

Amnion of Florida, based in Central Florida, is a leading provider of alternative medicine using cryopreserved placental cell transplants or allografts, processed from donated cellular birth tissue, which are natural alternatives to autologous regenerative medicine products. The primary function of our allogeneic regenerative treatments is to promote soft tissue joint/skin repair and regeneration mediated by growth factors and cells naturally found in placental tissue. These treatments have shown safety and efficacy in treating a variety of ailments including osteoarthritis, chronic ulcerative wounds, joint pain, skin rejuvenation, hair restoration, urinary incontinence, and ED. Learn more http://www.amnion.us.

More about Merakris Therapeutics, LLC

Merakris Therapeutics, based in Research Triangle Park, North Carolina, is focused on researching, developing, and marketing regenerative healthcare products. Merakris is pioneering commercially scalable biotherapeutic technologies derived from stem cells that have various clinical applications. Our vision is to improve global patient care and outcomes through the pioneering and innovation of acellular regenerative biotechnologies. Learn more at http://www.merakris.com.

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Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options - Business Wire

Mustang Bio Announces Updated Clinical Data on MB-107 Lentiviral Gene Therapy for Patients with X-Linked Severe Combined Immunodeficiency -…

MB-107 preceded by low-dose busulfan conditioning continues to be well tolerated and results in development of functional immune system in newly diagnosed infants with XSCID

Enhanced transduction procedure is demonstrating improvements in older patients with XSCID who received prior hematopoietic stem cell transplantation

Data presented by St. Jude Childrens Research Hospital and National Institutes of Health at 61st American Society of Hematology Annual Meeting

NEW YORK, Dec. 09, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced today that updated Phase 1/2 clinical data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID) were presented on Saturday by St. Jude Childrens Research Hospital (St. Jude) and today by the National Institutes of Health at the 61st American Society of Hematology (ASH) Annual Meeting.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude, and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, The updated clinical data presented at the 2019 ASH Annual Meeting underscore the curative potential of MB-107 for newly diagnosed infants with XSCID, as well as its meaningful impact on older XSCID patients who received prior hematopoietic stem cell transplantation. St. Jude recently received the 2019 Smithsonian Magazine American Ingenuity Award for development of the lentiviral gene therapy, highlighting its potential to have an impact on this devastating disease. We are excited to be working with St. Jude and NIH to advance MB-107 and look forward to transferring the IND from St. Jude to Mustang in the first quarter of 2020.

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study (Abstract Number: 2058)Poster presentation: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Childrens Research Hospital, Memphis, TN, USA

Interim data from the multicenter Phase 1/2 clinical trial for infants under the age of two treated with the lentiviral gene therapy preceded by low exposure-targeted busulfan conditioning were published in the New England Journal of Medicine. Updated data presented at the 2019 ASH Annual Meeting include three more patients (n=11), 8 months additional median follow up (23.6 months; range: 1.5 to 33.9 months), more extensive analysis of T and B cell functional recovery, and detailed vector integration site studies.

Data Highlights:

The results from treatment with low-dose busulfan conditioning and the novel lentiviral gene therapy in newly diagnosed infants with XSCID continue to be very promising, said Dr. Mamcarz. We are pleased that the therapy has been well tolerated and all patients with a follow up of more than 3 months recovered from pre-existing infections, are off protective isolation and prophylactic antimicrobials, and have normal growth in respect to height and weight. This reinforces our belief that the lentiviral gene therapy has the potential to be an attractive alternative to current XSCID therapies.

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency (Abstract Number: 608)Oral presentation: Harry Malech, M.D., Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA

Early outcome data for five older children and young adults with XSCID who received the lentivector (also known as lentiviral) gene therapy as salvage therapy after having previously received haplo-identical hematopoietic stem cell transplantation (HSCT) as infants without chemotherapy-based conditioning were previously reported and published in Science Translational Medicine. By 2016, three additional patients were treated, and the cohort of eight patients (referred to as Cohort A) has now been followed for 3 to 7 years. Among Cohort A, gradual clinical benefit in the clearance of chronic norovirus and associated improved abdominal complaints, malabsorption, growth and IgG production were observed, and four patients were able to cease immunoglobulin replacement therapy.

While the results were positive, the relatively inefficient transduction of hematopoietic stem/progenitor cells (HSPCs) required large quantities of vector. This resulted in relatively low VCN in myeloid cells in some patients, with delayed immune cell recovery and persistent clinical disease, especially in the last patient treated (patient 8). To address this, NIH developed a refined enhanced transduction (ET) procedure consisting of a single overnight transduction after 48 hours pre-stimulation in cytokines (Stem cell factor, Thrombopoietin, Flt3-ligand; 100ng/mL) and incorporated transduction enhancers LentiBoost 1:100 and dimethyl prostaglandin 2 (dmPGE2; 1uM).

The presentation at the 2019 ASH Annual Meeting included data from six patients (referred to as Cohort B) treated by NIH, including re-treatment of patient 8. The patients, who were aged 12 to 36, had significant problems with donor T cell infiltration of liver, bone marrow and kidneys, and were nearly absent of B and NK cells. The enhanced transduction procedure achieved much greater transduction efficiencies than were observed in Cohort A, with greater than 10-fold less vector, and resulted in faster immune reconstitution and more significant clinical benefit by 3 months.

We are encouraged by the significantly improved measures of early clinical outcomes from lentivector gene therapy in older children and young adults with XSCID using an enhanced transduction procedure with the addition of LentiBoost and dmPGE2, said Dr. Malech. Notably, we have seen an early appearance of B and NK cells at much higher levels in Cohort B than we previously observed in Cohort A, even at years after treatment. We look forward to continuing to closely monitor patients and report outcomes.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces Updated Clinical Data on MB-107 Lentiviral Gene Therapy for Patients with X-Linked Severe Combined Immunodeficiency -...

Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting – BioSpace

BOSTON and LONDON, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, will be presenting new registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, FL.

On Sunday, December 8, 2019, investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, on Monday, December 9, 2019, investigators will deliver an oral presentation featuring updated data from the ongoing clinical proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at SR-Tiget.

To learn more about Orchards approach to ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapy, conference attendees can visit booth #2228 in the Exhibition Hall.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time: Monday, December 9, 7:00am ET

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4 The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births in the United States and between one in 200,000 and one in 1 million in Europe.3 OTL-101 is an autologous, ex vivo, hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials in the United States and Europe, including a registrational trial at the University of California, Los Angeles (UCLA). OTL-101 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from the FDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5 There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.Id Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency. 2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314. 3Kwan A, et al. JAMA. 2014;312:729-738. 4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting - BioSpace

MacroGenics Presents Flotetuzumab Data in Patients with Primary Induction Failure and Early Relapsed Acute Myeloid Leukemia at the 2019 ASH Annual…

Rockville, MD, Dec. 09, 2019 (GLOBE NEWSWIRE) --

MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, today announced updated results from a Phase 1/2 dose expansion study of flotetuzumab, an investigational, bispecific CD123 x CD3 DART molecule in patients with primary induction failure and early relapsed acute myeloid leukemia (AML). The data were presented in an oral session at the 61st Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL, taking place December 7-10, 2019.

Patients with AML who have failed primary induction therapy or relapsed early after an initial response represent a significant unmet medical need. A remission rate of 32% observed in the ongoing study of flotetuzumab in this extremely challenging patient population is noteworthy, said Geoffrey Uy, M.D., Associate Professor, Department of Medicine, Division of Oncology at the Washington University School of Medicine in St. Louis. Importantly, by implementing a lead-in dosing schedule for flotetuzumab, as well as early intervention with tocilizumab in this study, we were able to mitigate cytokine release syndrome, known to be associated with T-cell engagers.

In the Phase 1/2 (NCT02152956) open-label, dose expansion study, 30 patients classified as primary induction failure or early relapsed AML who had received a median of four prior therapies were treated with flotetuzumab at the recommended phase 2 dose (RP2D) of 500 ng/kg/day by continuous infusion. Data were reported as of the cut-off date of November 1, 2019. The study is currently ongoing, with additional patients being enrolled.

Responses, including complete remission (CR), CRh (CR with partial hematological recovery) and CRi (CR with incomplete hematological improvement) per a modified International Working Group (IWG) Response Criteria for AML, are summarized in the table below. Four responders received allogeneic hematopoietic stem cell transplantation as consolidation therapy and remain in remission after 6 to 21 months.

The most common treatment-related adverse event (TRAE) was infusion-related reaction/cytokine release syndrome (IRR/CRS) that occurred in all (30/30) patients. However, most CRS events observed were of short duration and mild to moderate (grade 1 or 2) in severity, with only one grade 3 event reported in one patient.

Based on the encouraging data from this study, and pending anticipated discussions with the FDA in the first half of 2020, we are planning for a potential registration-enabling study of flotetuzumab in this high unmet need population of patients with refractory AML, who have limited treatment options, said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics.

A separate oral presentation described translational research that showed an inflammatory (IFN--related) gene expression signature in a subset of patients with AML that correlated with a lack of response to induction chemotherapy. Furthermore, the same gene signature was associated with patients more likely to respond to flotetuzumab, supporting the mechanism being exploited by this molecule. In addition, AML patients with an immune-infiltrated tumor micro-environment show high expression of immune checkpoint molecules, including PD-L1, which provides a scientific rationale for combining flotetuzumab with checkpoint blockade as a potential mechanism for enhanced anti-leukemic activity. MacroGenics has initiated a study combining flotetuzumab with MGA012, an anti-PD-1 antibody, given the strong preclinical and translational data that indicate the combination may enhance CD123-directed T cell killing.

Flotetuzumab Presentations at ASH

Oral Presentations

Poster Presentations

These slide and poster presentations are available on the Events & Presentations page on MacroGenics' website at http://ir.macrogenics.com/events.cfm.

Conference Call & Webcast

MacroGenics management and external guest speakers will host a conference call and audio webcast today at 8:00 p.m. ET to review the flotetuzumab data presented at the ASH Annual Meeting and discuss ongoing clinical development plans.

To participate in the MacroGenics ASH 2019 Conference Call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 3625435. A listen-only slide and audio webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company's website at http://ir.macrogenics.com/events.cfm. A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company's website for 30 days.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a hematopoietic stem cell malignancy characterized by differentiation arrest and uncontrolled clonal proliferation of neoplastic precursors that prevent normal bone marrow hematopoiesis. Nearly 20,000 new cases of AML are diagnosed in the U.S. each year, with a median age of 69 years at diagnosis. Approximately 40-50% of newly diagnosed patients fail to achieve a complete remission with intensive induction therapy (primary induction failure) or experience disease recurrence after a short remission duration (<6 months; early relapsed). A very small number of these patients are expected to respond to salvage therapy. In addition, although new targeted agents have been approved for the treatment of frontline or relapsed/refractory AML in recent years, approximately 50% of patients have no known targetable mutations.

About Flotetuzumab

Flotetuzumab (also known as MGD006) is a clinical-stage molecule that recognizes both CD123 and CD3. CD123, the interleukin-3 receptor alpha chain, has been reported to be over-expressed on cancer cells in AML and other hematologic malignancies. The primary mechanism of action of flotetuzumab is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cancer cells.

Flotetuzumab is currently being evaluated in the U.S. and Europe in a Phase 1/2 dose expansion study designed to assess the safety, tolerability, and initial anti-leukemic activity of the molecule in patients with relapsed/refractory AML. The U.S. Food and Drug Administration has granted orphan drug designation to flotetuzumab for the treatment of AML. A Phase 1/2 study in combination with MGA012, a proprietary anti-PD-1 monoclonal antibody, in patients with relapsed/refractory AML is being conducted ex-U.S. MGA012 (also known as INCMGA00012) was exclusively licensed to Incyte Corporation in 2017 under a global collaboration and license agreement; MacroGenics retains the right to develop its pipeline molecules with MGA012. MacroGenics retains global development and commercialization rights to flotetuzumab.

About MacroGenics, Inc.

MacroGenics is a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at http://www.macrogenics.com. MacroGenics, the MacroGenics logo and DART are trademarks or registered trademarks of MacroGenics, Inc.

Cautionary Note on Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the Company's strategy, future operations, clinical development of the Company's therapeutic candidates, milestone or opt-in payments from the Company's collaborators, the Company's anticipated milestones and future expectations and plans and prospects for the Company and other statements containing the words "subject to", "believe", "anticipate", "plan", "expect", "intend", "estimate", "project", "may", "will", "should", "would", "could", "can", the negatives thereof, variations thereon and similar expressions, or by discussions of strategy constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and enrollment of future clinical trials, expectations of expanding ongoing clinical trials, availability and timing of data from ongoing clinical trials, expectations for regulatory approvals, other matters that could affect the availability or commercial potential of the Company's product candidates and other risks described in the Company's filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views only as of the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so, except as may be required by law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date hereof.

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MacroGenics Presents Flotetuzumab Data in Patients with Primary Induction Failure and Early Relapsed Acute Myeloid Leukemia at the 2019 ASH Annual...

Olympic Hall of Fame Inductee Gary Hall Jr. Joins C3 International Board of Advisors – BioSpace

GARDEN GROVE, Calif., Dec. 9, 2019 /PRNewswire/ --C3International, a biopharmaceutical company that has played a leadership role in the emerging cannabinoid therapeutics health sector, today announced that Gary Hall, Jr. has joined the Board of Advisors for C3 International.

Gary Hall, Jr. is a healthcare-focused board member, patient advocate and key opinion leader with expertise in strategic alliance, marketing, clinical distribution, sports science, medical research and patient engagement. Hall has over 80 keynote presentations internationally, 10 Olympic medals in swimming and is an Olympic Hall of Fame Inductee. Using accomplishment in sports as a platform to advance diabetes advocacy for over 20 years, Gary amassed an influential network of policy makers, politicians, healthcare industry executives, diabetes care specialists, medical research, nutrition, philanthropic, sport, physical activity, obesity prevention, sports medicine and sports science organizations.

Gary has testified three times before Senate subcommittees on healthcare related issues and was a featured speaker at the 2016 Vatican hosted Cellular Horizons conference on stem cell therapy. He is the first recipient of USA Swimming's Humanitarian Award. One of his health community health initiatives was recognized at the Aspen Institute's Project Play Summit. He has presented at the American Heart Association, American Diabetes Association, American College of Sports Medicine, International Olympic Committee Medical Commission and National Youth Sports Health & Safety Institute conferences.

Idrasil is the first standardized form of medical cannabis. It offers all of the medicinal analgesic and therapeutic benefits of cannabis but is a superior alternative to opiates and life-threatening narcotics because physicians and caregivers can provide patients with safe, non-addictive, measurable dosages.

"The health benefits of CBD seem apparent and C3 International is the only company I've found that has Cannabinoid in a divisible, consistently dosed tablet," said Gary Hall Jr. "It's an honor to join the team. I look forward to providing perspective on potential athletic and healthcare applications. Idrasil aims to be the worldwide leader in medical cannabis."

"We are incredibly excited to welcome such an influential medical activist like Gary Hall Jr. onto our Board of Advisors," said Steele Clarke Smith III, Chairman and CEO of C3 International. "Hall will bring his vast expertise to our innovative biopharmaceutical company and we can't wait to work with him!"

Conditions treated with Idrasil include, but are not limited to, AIDS; anorexia; arthritis; autism; anxiety/depression; cancer; chronic pain; glaucoma; migraines; persistent muscle spasms; Parkinson's; seizures; severe nausea; Tourette's Syndrome, and any other chronic or a persistent medical symptoms that substantially limit major life activities as defined in the Americans with Disabilities Act of 1990.

Idrasil consists of a proprietary blend of concentrated cannabis extract that is 100% natural and organic. C3's proprietary process isolates all of the cannabinoids from the cloned cannabis plant, resulting in pure natural extraction in pill form to eliminate the unwanted euphoria and social risks associated with smoking cannabis products and unpredictable dosages of edible confections. Idrasil is a natural product that looks like any pill on the market.

Idrasil is aseptically processed and bacteria-free with a manufacturing process that is FDA-compliant. Idrasil is manufactured in a sterile ISO 9001 certified laboratory with a standardized and consistent 12.5mg, 25mg, or 100mg dose pill. Idrasil is categorized by the FDA as Generally Regarded As Safe (GRAS).

About Idrasil and C3 International

The mission of C3 International, Inc. is to manufacture the unique Intellectual Property for Idrasil, a major advancement in the standardization and administering of natural cannabinoids, in a tablet. Idrasil is a holistic alternative to addictive opiates and life-threatening narcotics, without euphoria. For more information on C3 International, Inc. visit http://www.c3internationalinc.com. To learn more about Idrasil, go to http://www.idrasil.com.

DISCLAIMER: THESE STATEMENTS HAVE NOT BEEN EVALUATED BY THE FOOD AND DRUG ADMINISTRATION. FURTHERMORE, NONE OF THE INFORMATION ON THIS PAGE SHOULD BE USED AS A SUBSTITUTE FOR THE ADVICE OF AN APPROPRIATELY QUALIFIED PHYSICIAN OR OTHER HEALTHCARE PROVIDER. THIS INFORMATION IS ONLY MEANT FOR INFORMATIONAL PURPOSES.

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Olympic Hall of Fame Inductee Gary Hall Jr. Joins C3 International Board of Advisors - BioSpace

Celgene Gave This Tech Back to Editas Medicine, but It Could Prove Valuable – The Motley Fool

In the middle of November, Editas Medicine (NASDAQ:EDIT) and Celgene (NASDAQ:CELG) announced changes to a development pact originally formed in 2015 with Juno Therapeutics, which is now part of Celgene. The agreement was amended in 2018, too, so the fact that changes were made wasn't necessarily big news. Editas received a $70 million upfront payment for executing the amended agreement, which was interpreted as the main takeaway from the announcement.

The announcement barely registered with investors and few gave it much thought for too long, especially after promising early results from the first clinical trials using a CRISPR-based medicine were announced by CRISPR Therapeutics days later.

But revisiting the amended collaboration agreement, and specifically what changes were made, hints at the long-term development plans of Editas Medicine. In short, it now has full control over an important class of immune cells. Whether that means the gene-editing pioneer lands another major development partner or goes full-steam ahead alone, investors can't overlook the significance.

Image source: Getty Images.

The basic scientific goal of the collaboration hasn't changed. Editas Medicine will use its gene-editing technology platform to engineer T cell receptors (TCR), while Juno Therapeutics will leverage its immunotherapy leadership to develop the engineered cellular medicines in clinical trials.

Why engineer TCRs? Immune cells rely on their receptors to identify targets, such as pathogenic bacteria and cancer cells. But immune cell receptors can be confused by molecules secreted within the tumor microenvironment, forcing them to halt their attack. They can also incorrectly attack an individual's own cells to trigger an autoimmune disease. A more recent concern stems from cellular medicines derived from a donor. Since the donor cells present different receptors compared to what the recipient's native T cells carry, the recipient's immune system (correctly) identifies the immunotherapy as a foreign substance, attacks it, and renders it less effective and less safe.

Therefore, it makes sense to engineer TCRs to create more potent and stealthier immunotherapies that are less likely to be tricked. Editas Medicine and Celgene still intend to do just that, albeit with subtle, yet important, differences to their development agreement.

Consideration

Previous Agreement (2015, 2018)

Amended Agreement (2019)

Focus

Cancer

Cancer and autoimmune diseases

Types of cells

CAR-T cells, alpha-beta T cells, gamma-delta T cells

Alpha-beta T cells

Juno Therapeutics exclusivity

Editas Medicine prohibited from all other work with CAR-T and TCRs in oncology

Editas Medicine prohibited from all other work on alpha-beta T cells and T cells derived from pluripotent stem cells

Upfront payment

$57.7 million (includes milestones collected under agreement)

$70 million

Milestone potential

$920 million plus tiered royalties

$195 million plus tiered royalties

Data source: SEC filings.

Essentially, Editas Medicine and Celgene have scaled back their original agreement in cancer and expanded their work to include autoimmune diseases. The most important detail is that the amended agreement allows the gene-editing pioneer to pursue the development of gamma-delta T cells, which were previously under the exclusive control of Juno Therapeutics. What does that mean?

Image source: Getty Images.

Without getting too far into the weeds, there are two main types of TCRs: alpha-beta and gamma-delta. The name refers to the molecular structure of the receptor, but that's not the important part.

Gamma-delta T cells, which comprise only about 5% of the T cells in your body, are thought to be one of the missing links in our understanding of the immune system. They're a mysterious bunch, but there could be significant value residing in the knowledge gaps.

These unique immune cells are governed by their own unique set of rules (relative to their alpha-beta peers) and straddle the innate immune system (what we're programmed with at birth) and adaptive immune systems (what's programmed as we encounter new environments throughout life). Gamma-delta T cells could be tinkered with in gut microbiome applications, to treat cardiovascular diseases, and to neutralize antibiotic-resistant infections. But the nearest commercial target of the mysterious immune cells is likely to be treating solid tumor cancers.

They possess potent anti-tumor activity where current immunotherapies fail, such as attacking cancer cells that lack tumor-specific antigens to target or that have become immune to checkpoint inhibitors. In fact, there's a link between certain cancer outcomes and the activity of specific gamma-delta T cells.

Given that, why would Celgene amend the agreement to ditch the rare subset of immune cells? Well, in August 2019, Celgene inked with a start-up called Immatics to develop engineered TCRs. The start-up's platform is based on gamma-delta tech.

Don't feel too bad for Editas Medicine, though. SEC filings reveal that the gene-editing pioneer didn't receive any money from the original collaboration deal with Celgene in the first nine months of 2019. That suggests the work had stalled or that the amendment was being hammered out for some time. The gene-editing pioneer wrestled back control of the tech and took a $70 million upfront payment to boot. While the potential milestone payments in the amended agreement are significantly lower than the originally promised bounty, Editas Medicine can offset that by signing a lucrative collaboration deal with a new partner.

There should be plenty of interest. Fellow gamma-delta T cell developer Adicet Bio recently landed an $80 million series B round funded in part by Johnson & Johnson, Regeneron,Samsung Biologics(not the same company as the electronics powerhouse), and Novartis. There's also Immatics, GammaDelta Therapeutics, and a handful of other start-ups making noise in the space.

Some competitors are directly engineering gamma-delta cells, and others are developing molecules to trigger the immune cells into action. Editas Medicine believes it has the edge, as it has a relatively precise and efficient method for engineering immune cells: gene editing.

The amended collaboration deal between Editas Medicine and Celgene received relatively little attention from investors. Perhaps that was a good thing, as Wall Street likely would have overreacted to the reduced scope of development and milestones. But investors that take the time to understand the details might be intrigued by the new research avenue for the gene-editing stock.

Can Editas Medicine become a leading force in gamma-delta T cell development? Perhaps. While it isn't the only company wielding a gene-editing platform, and CRISPR gene editing isn't the only type of gene editing, the company is well-positioned to take advantage of the opportunity. Investors will have to wait to see how (or if) the development strategy evolves around the new tech.

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Celgene Gave This Tech Back to Editas Medicine, but It Could Prove Valuable - The Motley Fool