Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 – PRNewswire

GAITHERSBURG, Md., Sept. 1, 2020 /PRNewswire/ –Cartesian Therapeutics, a fully integrated, clinical-stage biopharmaceutical company developing cell and gene therapies for cancer, autoimmune diseases and respiratory diseases, today announced that it has initiated a Phase 1/2 clinical trial of its lead RNA-engineered mesenchymal stem cell (MSC) therapy, Descartes-30, in patients with moderate-to-severe acute respiratory distress syndrome (ARDS), including that caused by COVID-19. Based upon the company’s research and analysis, this program is understood to be the first RNA-engineered cell therapy to enter clinical development for ARDS and COVID-19. It is also the first cell therapy to specifically degrade NETs, webs of extracellular DNA and histones that entrap inflammatory cells, block alveoli and vessels, and drive the pathogenesis of ARDS and COVID-19.

“Patients with ARDS, especially those with COVID-19 ARDS, generate copious amounts of NETs that physically obstruct alveoli and vessels, which leads to respiratory distress, immune-mediated thrombosis and a vicious cycle of inflammation,” said Bruce Levy, MD, Chief of Pulmonary and Critical Care Medicine at Brigham and Women’s Hospital and Parker B. Francis Professor at Harvard Medical School, and a clinical investigator in the Descartes-30 trial. “We would therefore expect that degrading NETs would improve oxygenation as well as resolve thrombi and quell inflammation in these patients. If successful, Descartes-30 would be a highly differentiated game-changer within our limited toolkit in managing this exceedingly difficult condition.”

Descartes-30 is an off-the-shelf (allogeneic) MSC product engineered with Cartesian’s RNA ArmorySM cell therapy platform. By expressing a unique combination of DNases that work synergistically, Descartes-30 can eliminate large, macroscopic amounts of NETs within minutes. MSCs are inherently immunomodulatory and naturally travel to the lungs, where they are expected to provide continuous, local delivery of DNases to NET-laden lung tissue.

“We engineered Descartes-30 without genomic modification, and therefore the production of DNases is expected to be time-limited to match the acute nature of ARDS,” said Metin Kurtoglu, MD, PhD, Chief Medical Officer at Cartesian. “Given thatDescartes-30will produce DNases locally and transiently, we anticipate that it will have a favorable benefit-to-risk profile. We also anticipate that these properties will enable Descartes-30 to treat a wide array of NET-related autoimmune and cardiovascular diseases.”

About the Phase 1/2a Clinical Trial

The “Phase 1/2a Study of Descartes-30 in Acute Respiratory Distress Syndrome” (NCT04524962) is enrolling patients with ARDS at multiple critical care units in the United States. Patients with ARDS due to COVID-19 are given enrollment priority. This first-in-human study aims to determine the safety and preliminary efficacy of Descartes-30 in patients with moderate to severe ARDS. The study, which is estimated to begin treatment in September, aims to enroll approximately 20 patients prior to initiation of a larger study. For more information visit

About ARDS and NETs

ARDS is a severe inflammatory lung disease with a mortality of over 40%. Inflammation leads to injury of lung tissue and leakage of blood and plasma into air spaces, resulting in low oxygen levels and often requiring mechanical ventilation. Inflammation in the lung may lead to inflammation elsewhere, causing shock and injury or dysfunction in the kidneys, heart, and muscles. Some causes of ARDS include COVID-19, severe pneumonia (including influenza), sepsis, trauma, and smoke inhalation.

NETs are inflammatory webs of DNA and proteins produced by neutrophils. NETs are commonly found in ARDS and are thought to exacerbate the disease by physically occluding air spaces and vessels, leading to reduced oxygenation and increased risk of immune thrombi. NETs are implicated in a variety of conditions beyond ARDS, including autoimmune and cardiovascular diseases.

About the RNA ArmorySM

The RNA ArmorySM is Cartesian’s proprietary RNA-based cell engineering platform that activates and arms cells with carefully selected, mRNA-based therapeutics. Unmodified donor cells enter the RNA ArmorySMin the millions; a battle-ready cell army leaves the RNA ArmorySMin the tens of billions. Each cell is equipped with a combination of therapeutics rationally chosen to have a synergistic effect on the disease. In the body, the cells deliver a precision-targeted treatment regimen directly to the site of disease. The cells express therapeutics with a defined half-life, enhancing their safety profile and making repeat dosing and outpatient administration possible. The platform is agnostic to cell type: we choose the best cell for the job, whether autologous or off-the shelf. For more information visit

About Cartesian Therapeutics

Founded in 2016,Cartesianis a fully integrated, clinical-stage biopharmaceutical company developing potent yet safer cell and gene therapies designed to benefit the broadest range of patients with cancer, autoimmune and respiratory diseases. Cartesianhas three products in clinical development under four open investigational new drug application (INDs) with the U.S. Food & Drug Administration (FDA). All investigational therapies are manufactured at Cartesian’s wholly owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD.Cartesian’s commanding IP position benefits in part from a broad, exclusive patent license from the National Cancer Institute. For more information visit

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Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 – PRNewswire

Mustang Bio Announces Orphan Drug Designation for MB-107 for the Treatment of X-linked Severe Combined Immunodeficiency in Newly Diagnosed Infants -…

September 02, 2020 08:00 ET | Source: Mustang Bio, Inc.

WORCESTER, Mass., Sept. 02, 2020 (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, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MB-107, Mustangs lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, in newly diagnosed infants under the age of two. The FDA previously granted Rare Pediatric Disease Designation in August 2020 and Regenerative Medicine Advanced Therapy designation in August 2019 to MB-107 for the treatment of XSCID in newly diagnosed patients. Additionally, the European Medicines Agency granted Advanced Therapy Medicinal Product classification to MB-107 in April 2020.

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, Mustang has had a productive quarter on the regulatory front. We are very pleased to achieve another significant milestone and receive Orphan Drug Designation for MB-107 for the treatment of XSCID in newly diagnosed patients. This designation for MB-107, in addition to its Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy designation and Advanced Therapy Medicinal Product classification, continues to enhance our regulatory pathway for a much-needed treatment option to address this devastating rare disease that affects children. We look forward to initiating our pivotal clinical programs for MB-107 in newly diagnosed infants with XSCID and MB-207 in previously transplanted patients with XSCID very soon.

MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital. In May 2020, Mustang submitted an investigational new drug application (IND) to the FDA to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are between two months to two years of age. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplantation (HSCT). The primary efficacy endpoint will be event-free survival. The initiation of this trial is expected early in the fourth quarter of 2020. Mustang is targeting topline data from this trial in the second half of 2022.

Another Phase 1/2 clinical trial for XSCID in patients over the age of two years, who have received prior HSCT, is underway at the National Institutes of Health, and Mustang expects to file an IND to the FDA to initiate a multi-center Phase 2 clinical trial in this population in the fourth quarter of 2020. This product candidate for XSCID in patients over the age of two years, who have received prior HSCT, is designated MB-207. The FDA granted a Rare Pediatric Disease Designation to MB-207 in August 2020.

About X-linked Severe Combined Immunodeficiency (XSCID) X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births. It is characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death by one year of age if untreated. Patients with XSCID have no T-cells or natural killer cells. Although their B-cells are normal in number, they are not functional. As a result, XSCID patients are usually affected by severe bacterial, viral or fungal infections early in life and often present with interstitial lung disease, chronic diarrhea and failure to thrive.

The specific genetic disorder that causes XSCID is a mutation in the gene coding for the common gamma chain (c), a protein that is shared by the receptors for at least six interleukins. These interleukins and their receptors are critical for the development and differentiation of immune cells. The gene coding for c is known as IL-2 receptor gamma, or IL2RG. Because IL2RG is located on the X-chromosome, XSCID is inherited in an X-linked recessive pattern, resulting in almost all patients being male.

About Mustang Bio Mustang Bio, Inc. 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 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 (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit

ForwardLooking StatementsThis 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 Begien Mustang Bio, Inc. (781) 652-4500

Investor Relations Contact: Daniel Ferry LifeSci Advisors, LLC (617) 430-7576

Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839

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Mustang Bio Announces Orphan Drug Designation for MB-107 for the Treatment of X-linked Severe Combined Immunodeficiency in Newly Diagnosed Infants -…

Animal Stem Cell Therapy Market Analysis 2020: Size, Share, Sales, Growth, Revenue, Type, Application & Forecast To 2026 – Bulletin Line

The recent report on GlobalAnimal Stem Cell Therapy Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 offered by Credible Markets, comprises of a comprehensive investigation into the geographical landscape, industry size along with the revenue estimation of the business. Additionally, the report also highlights the challenges impeding market growth and expansion strategies employed by leading companies in the Animal Stem Cell Therapy Market.

Impact of Covid-19 in Animal Stem Cell Therapy Market: Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost every country around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Animal Stem Cell Therapy market in 2020. The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor/outdoor events restricted; over forty countries state of emergency declared; massive slowing of the supply chain; stock market volatility; falling business confidence, growing panic among the population, and uncertainty about future.

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Key players in the global Animal Stem Cell Therapy market covered in Chapter 4:

ANIMAL CELL THERAPIES Celavet Animacel VETSTEM BIOPHARMA Cell Therapy Sciences Magellan Stem Cells Cells Power Japan Animal Care Stem Aratana Therapeutics VetCell Therapeutics MediVet Biologic U.S. Stem Cell J-ARM

In Chapter 11 and 13.3, on the basis of types, the Animal Stem Cell Therapy market from 2015 to 2026 is primarily split into:

Dogs Horses Others

In Chapter 12 and 13.4, on the basis of applications, the Animal Stem Cell Therapy market from 2015 to 2026 covers:

Veterinary Hospitals Research Organizations

Geographically, the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast (2015-2026) of the following regions are covered in Chapter 5, 6, 7, 8, 9, 10, 13:

United States, Canada, Germany, UK, France, Italy, Spain, Russia, Netherlands, Turkey, Switzerland, Sweden, Poland, Belgium, China, Japan, South Korea, Australia, India, Taiwan, Indonesia, Thailand, Philippines, Malaysia, Brazil, Mexico, Argentina, Columbia, Chile, Saudi Arabia, UAE, Egypt, Nigeria, South Africa and Rest of the World

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Some Points from Table of Content

Global Animal Stem Cell Therapy Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026

Chapter 1Report Overview

Chapter 2 Global Market Growth Trends

Chapter 3 Value Chain of Animal Stem Cell Therapy Market

Chapter 4 Players Profiles

Chapter 5 Global Animal Stem Cell Therapy Market Analysis by Regions

Chapter 6 North America Animal Stem Cell Therapy Market Analysis by Countries

Chapter 7 Europe Animal Stem Cell Therapy Market Analysis by Countries

Chapter 8 Asia-Pacific Animal Stem Cell Therapy Market Analysis by Countries

Chapter 9 Middle East and Africa Animal Stem Cell Therapy Market Analysis by Countries

Chapter 10 South America Animal Stem Cell Therapy Market Analysis by Countries

Chapter 11 Global Animal Stem Cell Therapy Market Segment by Types

Chapter 12 Global Animal Stem Cell Therapy Market Segment by Applications

Chapter 13 Animal Stem Cell Therapy Market Forecast by Regions (2020-2026)

Chapter 14 Appendix

The research provides answers to the following key questions:

What is the expected growth rate of the Animal Stem Cell Therapy market? What will be the market size for the forecast period, 2020 2026?

What are the major driving forces responsible for transforming the trajectory of the industry?

Who are major vendors dominating the Animal Stem Cell Therapy industry across different regions? What are their winning strategies to stay ahead in the competition?

What are the market trends business owners can rely upon in the coming years?

What are the threats and challenges expected to restrict the progress of the industry across different countries?

What are the key opportunities that business owners can bank on for the forecast period, 2020 2026?

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About Credible Markets

Credible Markets has emerged as a dependable source for the market research needs of businesses within a quick time span. We have collaborated with leading publishers of market intelligence and the coverage of our reports reserve spans all the key industry verticals and thousands of micro markets. The massive repository allows our clients to pick from recently published reports from a range of publishers that also provide extensive regional and country-wise analysis. Moreover, pre-booked research reports are among our top offerings.

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Thanks for reading this article you can also get individual chapter wise section or region wise report version like North America, Europe, MEA or Asia Pacific.

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Animal Stem Cell Therapy Market Analysis 2020: Size, Share, Sales, Growth, Revenue, Type, Application & Forecast To 2026 – Bulletin Line

Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market – The News Brok

New Study on the Global Stem Cell Therapy Market by PMR

Persistence Market Research recently published a market study that sheds light on the growth prospects of the global Stem Cell Therapy market during the forecast period (20XX-20XX). In addition, the report also includes a detailed analysis of the impact of the novel COVID-19 pandemic on the future prospects of the Stem Cell Therapy market. The report provides a thorough evaluation of the latest trends, market drivers, opportunities, and challenges within the global Stem Cell Therapy market to assist our clients arrive at beneficial business decisions.

As per the report, the global Stem Cell Therapy market is expected to grow at a CAGR of ~XX% during the stipulated timeframe owing to a range of factors including, favorable government policies, and growing awareness related to the Stem Cell Therapy , surge in research and development and more.

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Resourceful insights enclosed in the report:

Competitive Outlook

The competitive outlook section provides valuable information related to the different companies operating in the current Stem Cell Therapy market landscape. The market share, product portfolio, pricing strategy, sales and distribution channels of each company is discussed in the report.

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Prominent players covered in the report are:

Regional Assessment

The presented market study touches upon the market scenario in different regions and provides a deep understanding of the influence of micro and macro-economic factors on the prospects of the market in each region.

Some of the major companies operating in the global stem cell therapy market are Mesoblast Ltd., Celgene Corporation, Aastrom Biosciences, Inc. and StemCells, Inc.

Key points covered in the report

For any queries get in touch with Industry Expert @

The market report addresses the following queries related to the Stem Cell Therapy market:

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Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market – The News Brok

Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies – Business Wire

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and Metagenomi, a gene editing company discovering breakthrough systems for curing genetic disease, today announced that Vor will evaluate the potential use of Metagenomis gene editing technology to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia.

Cancer patients deserve therapies with strong effects on cancer cells and minimal effects on all other cells, said Tirtha Chakraborty, Ph.D., Vors VP and Head of Research. Our new partnership with Metagenomi will help us achieve this goal by engineering hematopoietic stem cells using precise yet flexible gene editing thereby ensuring that targeted therapies can live up to their name.”

The collaboration is non-exclusive and applies to pre-clinical research only. Further terms of the agreement are not being disclosed.

This partnership unites two transformative technologies our proprietary gene editing enzymes, and Vors platform for engineering hematopoietic stem cells such that they are inherently treatment-resistant, said Brian C. Thomas, Metagenomis CEO and co-founder. We are excited to be working together to bring both of these cutting-edge approaches into the clinic.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil.

About VOR33

Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, we believe that CD33 will become a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from acute myeloid leukemia.

About Metagenomi

Metagenomi is harnessing the vast information found in life on Earth to develop cures for genetic disease. Using proprietary data collected from around the world, Metagenomi has developed novel gene editing tools that enable next-generation gene and cell therapies.

Metagenomi is based out of Emeryville, California, and was founded by pioneers in the field of metagenomics, Jill Banfield and Brian C. Thomas. Metagenomi generates massive quantities of data from natural environments, producing complete genomes from organisms that are otherwise unknown. Metagenomi then unlocks the information captured in these genomes to develop game-changing in vivo and ex vivo therapeutics.

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Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies – Business Wire

Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I – BioSpace

Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) — Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today’s results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. “These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

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 investigationalex vivoautologous hematopoietic stem cell 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 theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Ourex vivoautologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (, the investor relations website (, and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This 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, plans, 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, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, 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. 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, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its 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 or that long-term adverse safety findings may be discovered; 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 or 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 quarterly report on Form 10-Q for the quarter endedJune 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. 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.


Investors Renee Leck Director, Investor Relations +1 862-242-0764

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378

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Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I – BioSpace

New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem…

Impact of New Tissue Stem Cell Counting Algorithms

BOSTON (PRWEB) September 01, 2020

Stem cell biotechnology company, Asymmetrex, has been counting tissue stem cells like those used for bone marrow and cord blood transplantation therapies for a few years now. Recently, the company announced the issue of patents for its first-in-kind technology both in the U.S. and the U.K. However, until last Friday, August 28, Asymmetrex had not reported in the peer-reviewed academic literature how it achieves this feat that had been pursued by many distinguished labs for more than six decades.

Now in a report published in a special issue of OBM Transplantation, a peer-review journal for transplantation medicine research, Asymmetrex completes its introduction of the new technology to the fields of stem cell science and stem cell medicine. The report is the second invited article published in a special issue focused on the Isolation and Characterization of Adult Therapeutic Cells.

The new report describes Asymmetrexs discovery of mathematical formulas, call algorithms, that can be used to determine the number of stem cells in complex tissue cell preparations, like experimental samples or patient treatments. The stem cell counting algorithms are specific for different types of tissue stem cells. So, the algorithms defined for blood stem cells are distinct from the algorithms for liver stem cells, or lung stem cells. Once an algorithm is defined by the Asymmetrex technology, it can be used repeatedly as a simple, rapid, and inexpensive test to determine the quantity and dosage of its specific tissue stem cell type.

Asymmetrexs founder and director, James L. Sherley, M.D., Ph.D., anticipated the August publication of the new algorithms in a talk given earlier at the 6th Annual Perinatal Stem Cell Society Congress in March of this year. Then and now, he says that he believes, Now that the tissue stem cell counting algorithms are available, everything will change in stem cell science and medicine.

Prior to Asymmetrexs technology, there was no method for counting tissue stem cells in research, medicine, or for any other of their many uses. So, the impact of the stem cell counting algorithms in research and medicine is far-reaching. Such information is a game changer for accelerating progress in stem cell science and stem cell medicine, including improving treatments like gene therapy whose success depends on targeting tissue stem cells. There will also be tremendous gains in cell biomanufacturing, drug development, and environmental toxicology, all whose capabilities are currently limited by the lack of a facile means to quantify tissue stem cells.

To make the new counting technology readily accessible for evaluation by the greater academic, medical, and industrial stem cell communities, Asymmetrex provides free tissue stem cell counting on its company website.

About Asymmetrex

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. The companys U.S. and U.K. patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of effective use of human adult tissue stem cells for regenerative medicine and drug development. Asymmetrex markets the first technology for determination of the dose and quality of tissue stem cell preparations (the AlphaSTEM Test) for use in stem cell transplantation therapies and pre-clinical drug evaluations. Asymmetrex is a member company of the Advanced Regenerative Manufacturing Institute BioFabUSA and the Massachusetts Biotechnology Council.

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New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem…

FDA Approves Azacitidine tablets for Continued Treatment of AML – Cancer Network

The FDA has approved 300 mg tablets of azacitidine (Onureg) for the continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy, according to Bristol Myers Squibb, the developer of the agent.

The approval was based on results from the pivotal phase 3 QUAZAR AML-001 study in which treatment with azacitidine tablets resulted in a statistically significant and clinically meaningful improvement in overall survival (OS), the studys primary end point, of almost 10 months compared to placebo. Results from the study were first presented at the American Society of Hematology (ASH) Annual Meeting in December 2019.

Continued treatment with [azacitidine tablets]demonstrated an overall survival benefit in adults with AML who had achieved first complete remission in the QUAZARAML-001 study and, notably, it has the potential to do this in a convenient manner, given its once daily oral formulation, Andrew Wei, MBBS, PhD, QUAZAR AML-001 lead investigator from Alfred Hospital and Monash University in Melbourne, Australia, said in a press release. This approval should help establish continued treatment with [azacitidine tablets] as a standard component of AML therapy for adults who achieved first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant.

The phase 3, international, randomized, double-blind study enrolled patients who were 55 years or older, had AML, were within 4 months of achieving first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry), and were not candidates for hematopoietic stem cell transplant (HSCT) at the time of screening. Overall, the study enrolled 472 patients who were randomized 1:1 to receive either 300 mg of azacitidine tablets (n = 238) or placebo (n = 234) orally, once daily, for 14 days of a 28-day cycle, plus best supportive care.

Median OS from time of randomization was greater than 2 years (24.7 months; 95% CI, 18.7-30.5) among patients who received azacitidine tablets compared to 14.8 months (95% CI, 11.7-17.6) among patients receiving placebo (HR, 0.69; 95% CI, 0.55-0.86; P = .0009).Notably, the azacitidine tablets were continued until disease progression or unacceptable toxicity.

Serious adverse events (AEs) occurred in 15% of patients who received azacitidine tablets. Serious AEs observed in at least 2% of patients who received the agent included pneumonia (8%) and febrile neutropenia (7%). Only 1 fatal adverse reaction (sepsis) occurred in a patient who received azacitidine tablets.

The most common adverse events observed with azacitidine tablets versus placebo were nausea (65% vs 24%, respectively), vomiting (60% vs 10%), diarrhea (50% vs 21%), fatigue/asthenia (44% vs 25%), constipation (39% vs 24%), pneumonia (27% vs 17%), abdominal pain (22% vs 13%) arthralgia (14% vs 10%), decreased appetite (13% vs 6%), febrile neutropenia (12% vs 8%), dizziness (11% vs 9%), and pain in extremity (11% vs 5%). Moreover, of those who received azacitidine, permanent discontinuation due to an AE occurred in 8% of patients.

The FDA approval of [azacitidine tablets] is the culmination of over a decade of research and 13 pre-clinical and clinical trials. We are grateful to the patients, families and caregivers who participated in and supported these trials, and who ultimately made todays advancement possible, Giovanni Caforio, MD,chairman and chief executive officer, Bristol Myers Squibb, said in the release. This milestone is representative of our commitment to helping patients with hard-to-treat cancers live longer, and the approval of [azacitidine tablets] as an oral therapy option for patients is more relevant now than ever as the world continues to navigate the COVID-19 pandemic.

Importantly, Bristol Myers Squibb indicated that azacitidine tablets have warnings and precautions for risks of substitution with other azacitidine products, myelosuppression, increased early mortality in patients with myelodysplastic syndromes (MDS) and embryo-fetal toxicity. Due to substantial differences in the pharmacokinetic parameters, the company indicated azacitidine tablets should not be substituted for intravenous or subcutaneous azacitidine as it may result in a fatal adverse reaction.

The new drug application for this indication was previously granted priority review designation by the FDA, and a marketing authorization application for this indication was validated by the European Medicines Agency in May 2020.


U.S. Food and Drug Administration Approves Onureg (azacitidine tablets), a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia [news release]. Princeton, NJ. Published September 1, 2020. Accessed September 1, 2020.

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FDA Approves Azacitidine tablets for Continued Treatment of AML – Cancer Network

Prior Use of Novel Agents Does Not Affect alloHCT Outcomes in CLL – Cancer Therapy Advisor

The number of prior novel agents administered or their use immediately prior to allogeneic hemopoietic stem cell transplant (alloHCT) for the treatment of chronic lymphocytic leukemia (CLL) was not associated with outcomes, according to results of a retrospective study published in Blood Advances.1

Novel agents such as BCR, BTK, PI3K, and BCL2 inhibitors have generally improved outcomes among patients with CLL, but a subset of patients experience disease progression, resistance, high-grade transformation, or drug intolerance and require additional curative modalities.

Understanding outcomes for potentially curative modalities including alloHCT following novel agent therapy is critical while devising treatment sequences aimed at long-term disease control, the authors wrote.

This multicenter, retrospective cohort study included 65 patients with CLL who underwent alloHCT. All patients had received at least 1 novel agent prior to their transplant. Patient-related and disease-related characteristics were assessed, as well as prior treatment and transplant characteristics.

At baseline, the median patient age was 50 years at CLL diagnosis and 60 years at alloHCT. The HCT-comorbidity index (CI) was 0 among 38% of patients, 1-2 among 37%, and 3 among 25%. The majority of patients received a reduced-intensity conditioning regimen and a calcineurin inhibitor with methotrexate as graft-versus-host disease (GVHD) prophylaxis. The median number of previous lines of therapy was 3, with a median of 1 prior novel agent.

At 24 months, the progression-free survival (PFS) was 63% and overall survival (OS) was 81%. Nonrelapse mortality was 13% and relapse occurred among 27% of patients.

The number of novel agents used prior to transplant and the timing of their use was not associated with PFS or OS. In univariate analyses, there was no association between PFS and prior use of ibrutinib, venetoclax, PI3K inhibitors, 2 novel agents, or novel agent use immediately before alloHCT. Results were similar for OS.

Poor-risk characteristics, complete versus partial remission, and transplant characteristics were also not associated with PFS after transplant.

Multivariate analysis, however, demonstrated that HCT-CI 1 was significantly associated with PFS (hazard ratio, 3.3; 95% CI, 1.1-9.9; P =.035).

The authors concluded that prior novel agents do not appear to impact the safety of alloHCT, and survival outcomes are similar regardless of number of novel agents received, prior chemoimmunotherapy exposure, or novel agent immediately preceding alloHCT.


Roeker LE, Dreger P, Brown JR, et al. Allogeneic stem cell transplantation for chronic lymphocytic leukemia in the era of novel agents. Blood Adv. 2020;4:3977-3989. doi:10.1182/bloodadvances.2020001956

Prior Use of Novel Agents Does Not Affect alloHCT Outcomes in CLL – Cancer Therapy Advisor

COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle –

In mid-August, the US Food and Drug Administrations (FDA) Oncologic Drugs Advisory Committee voiced support for the efficacy of remestemcel-L in children with steroid-resistant graft-versus-host (GvHD) disease. The positive vote moved Mesoblast a step closer to winning approval for the mesenchymal stem cell therapy.

While seeking approval in that long-targeted indication, Mesoblast is also working to show the cell therapy is effective in patients with acute respiratory distress syndrome (ARDS) caused by infection with SARS-CoV-2.

Talking to investors on a fourth quarter results conference call late last week, Mesoblast CEO Silviu Itescu explained how the potential size of the COVID-19 market opportunity creates a substantial challenge.

We … have to be prepared to substantially scale up manufacturing … to be in a position next year to make sufficient quantity of product to start to meet some of this unmet need. We are able to implement proprietary xeno-free technologies and we certainly have plans to move into 3D bioreactors to allow us to have sufficient capability to meet this large unmet need, said Itescu.

Like many organizations targeting COVID-19, including groups such as AstraZeneca and Regeneron Pharmaceuticals that have large in-house operations, Mesoblast is planning to partner to gain the scale needed to manufacture the quantities of remestemcel-L it may need.

Mesoblast is currently running a phase 3 trial of remestemcel-L in ARDS. Itescu is assuming that the company will be entering into a strategic partnership for manufacturing commercialization to serve the ARDS indication.

The need for remestemcel-L in ARDS will depend on the progress of COVID-19 vaccines, which could significantly reduce the number of people suffering the complication of SARS-CoV-2 infection, and the strength of the phase 3 data.

Itescu explained the rationale for developing remestemcel-L in COVID-19 on the conference call. Like GvHD, a hyperactive immune response, known as a cytokine storm, is implicated in ARDS. In ARDS the cytokine storm manifests in severe inflammation of the lungs.

Remestemcel-L has shown anti-inflammatory effects during its development in GvHD. In addition, there is evidence the cell therapy migrates to the lungs after intravenous administration, suggesting it will accumulate in the part of the body where it is needed most in ARDS,

US physicians administered remestemcel-L to ventilator-dependent patients under a compassionate use program earlier this year. Nine of the 12 patients were taken off ventilator support, after 10 days in median, and later discharged from the hospital.

The evidence to support the use of remestemcel-L in COVID-19 led Mesoblast to start a 300-subject clinical trial. Mesoblast is assessing the effect of remestemcel-L on mortality after 30 days and is set to hold a series of interim analyses as increasing percentages of participants reach that point. If the data link remestemcel-L to improved survival, Mesoblast will seek expedited regulatory approval.

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COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle –