ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition, which will take place virtually from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.
An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH, including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.
CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.
The accepted abstract is now available on the ASH conference website.
About CTX001 CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.
Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.
About CLIMB-111 The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About CLIMB-121 The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About the Gene-Editing Process in These Trials Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.
About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.
CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data and plans to present data at the annual ASH meeting and exposition; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.
About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to present data at the annual ASH meeting and exposition, the development, including expected timeline for development, and potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
CRISPR Therapeutics Investor Contact: Susan Kim, +1 617-307-7503 firstname.lastname@example.org
CRISPR Therapeutics Media Contact: Rachel Eides WCG on behalf of CRISPR +1 617-337-4167 email@example.com
Vertex Pharmaceuticals Incorporated Investors: Michael Partridge, +1 617-341-6108 or Zach Barber, +1 617-341-6470 or Brenda Eustace, +1 617-341-6187
Media: firstname.lastname@example.org or U.S.: +1 617-341-6992 or Heather Nichols: +1 617-839-3607 or International: +44 20 3204 5275
- Co-founder Thomas Smeenk on the benefits of Hemostemix autologous stem cell therapy - InvestorIntel - January 12th, 2021
- Covid-19 Impact On Stem Cell Therapy Market 2020 Huge Potential in Future Growth by 2027 | Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc.,... - January 12th, 2021
- Adipose Derived Stem Cell Therapy Market Analysis and Forecast, 2020-2026 Coherent Market Insights | BioRestorative Therapies, Inc., Celltex... - January 12th, 2021
- New COVID-19 Treatment Uses Stem Cells - Freethink - January 12th, 2021
- Creative Medical Technology Holdings Files Investigational New Drug Application (IND) with FDA for Treatment of Stroke using ImmCelz Regenerative... - January 12th, 2021
- Global Cell Freezing Media for Cell Therapy Market 2020 Dynamics Analysis, Size Witness Growth Acceleration During 2025 - KSU | The Sentinel Newspaper - January 12th, 2021
- Gamida Cell to Present Full Data from Phase 3 Study of Omidubicel at TCT, the Combined Transplantation and Cellular Therapy Meetings of ASTCT and... - January 12th, 2021
- Cate Dyer of StemExpress is Named Businesswoman of the Year! - PRNewswire - January 12th, 2021
- Adocia Files Patent on a Hydrogel Scaffold for Cell Therapy in the Treatment of Type 1 Diabetes - Business Wire - January 12th, 2021
- Gene Therapy Market Estimated to Record Highest CAGR by 2024 - KSU | The Sentinel Newspaper - January 12th, 2021
- Stem cell treatment corrects skull shape and restores brain function in mouse model of childhood disorder - National Institutes of Health - January 9th, 2021
- Doctors Make Medical Breakthrough In Treating Severe Cases Of COVID - CBS New York - January 9th, 2021
- Global Stem Cell Therapy Market Report 2020: Market to Recover and Reach $14.76 Billion in 2023 at a CAGR of 19.62% - Forecast to 2030 - Yahoo Finance - January 9th, 2021
- Engineered stem cells that evade immune detection could boost cell therapy and I-O - FierceBiotech - January 9th, 2021
- BENEV Announces Investigative Report on Combination Treatment with Human Adipose Tissue Stem Cell- derived Exosomes and Fractional CO2 Laser for Acne... - January 9th, 2021
- Four promising COVID-19 therapies being tested at nearby UVA - Rappahannock News - January 9th, 2021
- MCL Landscape Adapts to Changes After CAR T-Cell Therapy Approval - OncLive - January 9th, 2021
- Regenerative Medicine Market to Reach Valuation US$ 23.7 Bn by 2027 - GlobeNewswire - January 9th, 2021
- Unlocking The Unlimited Potential Of Stem Cells - CodeBlue - January 9th, 2021
- Stem Cell Treatment Proving Highly Successful In Treating Most Severe Cases Of COVID - Yahoo News - January 9th, 2021
- Factor Bioscience spins out a new cell therapy player with eyes on the clinic within 2 years - Endpoints News - January 9th, 2021
- Comparison of the efficiency, safety, and survival outcomes in two stem cell mobilization regimens with cyclophosphamide plus G-CSF or G-CSF alone in... - January 9th, 2021
- Top 10 ALS Stories of 2020 - ALS News Today - January 9th, 2021
- Celularity and GX Acquisition Corp. Announce Merger Agreement to Create a Publicly Listed Leader in Allogeneic Cellular Therapy - BioSpace - January 9th, 2021
- Results Show Promising Efficacy in MCL With Next-Generation BTK Inhibitor LOXO-305 - Cancer Network - January 9th, 2021
- BlueRock Therapeutics in Collaboration with Memorial Sloan Kettering Cancer Center Receives IND Clearance for DA01 in Parkinson's Disease - BioSpace - January 9th, 2021
- Stem Cell Therapy Market Size, Share & Trends Analysis Report By Product Types, And Applications Forecast To 2026 - Farming Sector - January 5th, 2021
- Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2027 - LionLowdown - January 5th, 2021
- Stem Cell Therapy Market Analysis, COVID-19 Impact,Outlook, Opportunities, Size, Share Forecast and Supply Demand 2021-2025 - Farming Sector - January 5th, 2021
- AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics' Newly Approved Libmeldy - PRNewswire - January 5th, 2021
- Forge Biologics Announces FDA Clearance of Investigational New Drug Application for Phase 1/2 Clinical Trial (RESKUE) of FBX-101 Gene Therapy for... - January 5th, 2021
- MorphoSys and Incyte Announce the Acceptance of the Swissmedic Marketing Authorization Application f - PharmiWeb.com - January 5th, 2021
- Healthcare resource utilization and costs among patients with relapsed and/or refractory multiple myeloma treated with proteasome inhibitors in... - January 5th, 2021
- Global Circulating Tumor Cells and Cancer Stem Cells Market To Reach A New Threshold of Growth By 2026 - The Courier - January 5th, 2021
- Catheters Market: Europe and North America Emerge as Leading Regional Market with Well-established Healthcare Infrastructure - BioSpace - January 5th, 2021
- The Worldwide Cell Isolation/Cell Separation Industry is Expected to Grow at a CAGR of 16.8% Between 2020 to 2025 - PRNewswire - January 5th, 2021
- Near-infrared oxidative phosphorylation inhibitor integrates acute myeloid leukemiatargeted imaging and therapy - Science Advances - January 5th, 2021
- Global Cell Therapy Market Report 2020: Market to Recover in 2023 - PRNewswire - January 3rd, 2021
- Lonza's Cocoon will soon make dozens of stem cell therapies all at once - Innovation Origins - January 3rd, 2021
- Induced Pluripotent Stem Cell Derived Human Lung Organoids to Map and Treat the SARS-CoV2 Infections In Vitro - DocWire News - January 3rd, 2021
- Creative Medical Technology Holdings Announces Patent filing based on Preclinical Data in Model of Heart Attack using ImmCelz Regenerative... - January 3rd, 2021
- Haywards Heath carer trying to raise funds for MS treatment - The Argus - January 3rd, 2021
- Chronic graft-versus-host disease in children and adolescents with thalassemia after hematopoietic stem cell transplantation - DocWire News - January 3rd, 2021
- COVID-19-positive cancer patients undergoing active anticancer treatment: An analysis of clinical features and outcomes - DocWire News - January 3rd, 2021
- Cell Therapy Market 2020-2026 by Growth, Demand and Upcoming Business Opportunities - Farming Sector - January 3rd, 2021
- Rheumatoid Arthritis Stem Cell Therapy Market Latest Trends and Future Growth Study by 2029 - Farming Sector - December 31st, 2020
- Creative Medical Technology Holdings Announces Reversion of Liver Failure Using ImmCelz Personalized Cellular Immunotherapy in Preclinical Model -... - December 31st, 2020
- Impacts of COVID 19 on Stem Cell Therapy Market 2020 Size, Demand, Opportunities & Forecast To 2026 - Factory Gate - December 31st, 2020
- Efficacy and safety of mesenchymal stem cells for the treatment of patients infected with COVID-19: a systematic review and meta-analysis protocol -... - December 31st, 2020
- Global Stem Cell Therapy Market Industry 2021 In-depth Market Analysis and Recent Developments, Share, Revenue and Forecast 2025 | Anterogen Co. Inc.... - December 31st, 2020
- Nociceptive neurons shown to boost hematopoiesis | 2020-12-28 - BioWorld Online - December 28th, 2020
- Stem Cell Therapy Market 2020: Demand Analysis, Key Players, Trends, Sales, Supply, Deployment Model, Segments, Organization Size, Production, Revenue... - December 28th, 2020
- Stem Cell Therapy Market (COVID-19 Analysis) 2020-2025 Is Booming Worldwide with Comprehensive Study Explores Huge Revenue Scope in Future - Farming... - December 28th, 2020
- Humanigen Secures US Patent for Lenzilumab in Preventing Cytokine Storm and Neurotoxicity Related to CAR-T Cell Therapy - BioSpace - December 28th, 2020
- Immunotherapy Continues on Positive Trajectory in Lymphoid Malignancies - OncLive - December 28th, 2020
- Stem Cell Therapy Market Size 2020 | Opportunities, Regional Overview, Top Leaders, Revenue and Forecast to 2025 - LionLowdown - December 28th, 2020
- Adipose Derived Stem Cell Therapy Market 2020 Analysis, Growth, Size, Shares, Drivers, Advancements, Challenges, Sophisticated Demand & Key... - December 28th, 2020
- Stem cells from cord blood can now be used across many conditions: Mayur Abhaya, MD & CEO, LifeCell Internat.. - ETHealthworld.com - December 28th, 2020
- Comprehensive Report on NK Cell Therapy and Stem Cell Therapy Market 2021 | Size, Growth, Demand, Opportunities & Forecast To 2027 | Chipscreen... - December 28th, 2020
- The Top 5 Most-Read Oncology Stories of 2020 - AJMC.com Managed Markets Network - December 28th, 2020
- Adipose Tissue-derived Stem Cell Therapy Market Share Analysis, Application, Strategies of Key Players & Forecast to 2025 - Factory Gate - December 28th, 2020
- Study clarifies the impact of getting old on hematopoietic stem cells - Microbioz India - December 28th, 2020
- Report On Canine Stem Cell Therapy Market to 2026: (Industry Insights, Company Overview and Investment Analysis) - Farming Sector - December 24th, 2020
- Six Benefits of All-on-4 Implants Compared to Regular Dentures - Magazine of Santa Clarita - December 24th, 2020
- Allogeneic SCT Benefits Children and Adolescents With Relapsed Anaplastic Large Cell Lymphoma - OncLive - December 24th, 2020
- Stem cells in the brain remain active and divide over months - News Landed - December 24th, 2020
- Canine Stem Cell Therapy Market 2026 by Product Type, Applications, Manufacturers, Revenue, Price, Share and Growth Rate & Forecast -... - December 24th, 2020
- Exclusive Research on Stem Cell Therapy Products Market 2020 Segments, Regions, Strategy Analysis: Mesoblast, TiGenix, Osiris - LionLowdown - December 24th, 2020
- Heterogenous Nature of MDS Warrants Further Research, Review Finds - AJMC.com Managed Markets Network - December 24th, 2020
- Stem Cell Therapy Shows 2-year Benefit for Progressive MS Patients in Phase 1 Trial - Multiple Sclerosis News Today - December 19th, 2020
- Dr Clarence Moore Discusses the Importance of Stem Cell Therapy, Heart Health in Beta Thalassemia - AJMC.com Managed Markets Network - December 19th, 2020
- For Patients With HMA-Resistant MDS, What Are Their Options? - AJMC.com Managed Markets Network - December 19th, 2020
- The Adrenomyeloneuropathy Treatment Market to grow on an emphatic note from 2019 to 2029 - PharmiWeb.com - December 19th, 2020
- Are Hiccups a Sign of the New Coronavirus? - Healthline - December 19th, 2020
- Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On - Good News Network - December 19th, 2020
- Therapeutic Solutions International Files Investigational New Drug Application (IND) with FDA for Treatment of Chronic Traumatic Encephalopathy Using... - December 18th, 2020
- Physio Logic Brings Cutting-edge Regenerative Treatments for Sport Injuries and Arthritis to New York City - PRNewswire - December 18th, 2020
- AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy... - December 18th, 2020
- ONK Therapeutics Announces Three Exclusive Option License Agreements, Which Extend and Strengthen its Dual-Targeted NK Cell Therapy Pipeline -... - December 18th, 2020
- Mesoblast shares fall on trials mixed results with heart injection treatment - Stockhead - December 18th, 2020