Findings suggest motixafortide alone, and in combination with natalizumab, could support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle - Data from proof-of-concept study shows that motixafortide was safe and well tolerated Oral presentation at ASH 2024 on Saturday, December 7, 2024 in San Diego, California TEL AVIV, Israel and...Read more
Initial Results from BEACON Phase 1/2 Clinical Trial Demonstrate Potential for Differentiation of Base Editing and BEAM-101 Preclinical ESCAPE Data Establish Proof-of-concept for Non-genotoxic, Antibody-based Conditioning and Engraftment in Non-human Primates Clinical Data from Phase 1/2 BEAM-201 Trial Demonstrate Therapeutic Potential of First Quadruplex-edited Allogeneic CAR-T Cell Therapy Beam to Host Investor Event on Dec. 8,...Read more
Topline Data from 52-week Phase 3 Study Expected in Late 2025 CAMBRIDGE, Mass., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that enrollment is complete for the Phase 3 RISE UP study. This global, double-blind, randomized, placebo-controlled trial is evaluating the efficacy and...Read more
First regulatory authorization of a CRISPR-based gene-editing therapy in Canada TORONTO, Sept. 25, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced Health Canada has granted Marketing Authorization for PrCASGEVY® (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease...Read more
SUNNYVALE, Calif., Sept. 12, 2024 (GLOBE NEWSWIRE) -- 23andMe Holding Co. (Nasdaq: ME) (23andMe), a leading genetic health and biopharmaceutical company, in collaboration with lead researchers at National Human Genome Research Institute, part of the National Institutes of Health (NIH), and Johns Hopkins University, conducted one of the largest and most diverse studies on sickle cell trait (SCT). Many prior SCT research studies have only...Read more
Sickle cell disease affects approximately 100,000 patients in the U.S. annually and is a debilitating and life-threatening condition with high unmet need Current conditioning with non-targeted chemotherapies provides limited access to potentially curative bone marrow transplant and recently approved gene therapies for sickle cell disease patients Initial trial focused on conditioning for bone marrow transplant intended to...Read more
Results from CLIMB-111, -121 and -131 accepted for oral presentation Data from these trials, with the longest follow-up of more than five years, demonstrate transformative, consistent and durable benefit of CASGEVY™ Safety profile consistent with busulfan conditioning and autologous hematopoietic stem cell transplant BOSTON / Jun 14, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term...Read more
All patients treated in the RUBY trial are free of vaso-occlusive events post-renizgamglogene autogedtemcel (reni-cel) infusion Patients had early normalization of total hemoglobin with a mean within the normal range at >14 g/dL and rapid and sustained improvements in fetal hemoglobin well above levels of >40%. Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan...Read more
MADRID, June 14, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported data highlighting its optimized, closed and automated manufacturing process for its base-edited CD34+ hematopoietic stem and progenitor cell (HSPC) genetic medicines in a poster presentation at the European Hematology Association (EHA) Hybrid Congress. The...Read more
NEW YORK, June 12, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a scientific article in Nature Communications, unveiling a non-viral gene therapy approach for sickle cell disease. Sickle cell disease (SCD) is one of...Read more
SOMERVILLE, Mass. / May 06, 2024 / Business Wire / bluebird bio, Inc. (Nasdaq: BLUE) announced today that it has completed the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a one-time gene therapy for the treatment of sickle cell disease in patients with a history of vaso-occlusive events. LYFGENIA was approved by the FDA in December 2023 and is the most deeply studied gene therapy for sickle cell disease, with...Read more
SOMERVILLE, Mass. / Mar 11, 2024 / Business Wire / bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio”) today announced it has signed its first Medicaid outcomes-based agreement for LYFGENIA™ (lovotibeglogene autotemcel, also known as lovo-cel) with the state of Michigan. LYFGENIA is a one-time gene therapy approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events (VOEs)....Read more
Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia may be eligible for treatment LONDON / Feb 13, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved...Read more
CASGEVY™ is first medicine ever to be evaluated through the SFDA’s Breakthrough Medicines Program First treatment center in Saudi Arabia has been activated BOSTON / Jan 09, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the Saudi Food and Drug Authority (SFDA) granted Marketing Authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the...Read more
Proof-of-concept study is initial step toward goal of identifying more efficient CD34+ HSC mobilization regimen for patients with sickle cell disease choosing gene therapy TEL AVIV, Israel, Dec. 21, 2023 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that the first patient has been...Read more
If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be eligible for treatment Approval decision from the European Commission is expected in Q1 2024 BOSTON / Dec 15, 2023 / Business...Read more
Longer-term follow-up data demonstrate consistent and durable response to treatment BOSTON / Dec 11, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced two oral presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition from the global pivotal trials of CASGEVY™ (exagamglogene autotemcel [exa-cel]). Data from 96 patients (44 sickle cell disease [SCD], 52...Read more
Treatment with Mitapivat Demonstrated Statistically Significant Improvement in Hemoglobin Response Compared to Placebo Improvements Observed in Annualized Rates of Sickle Cell Pain Crises, Markers of Hemolysis and Erythropoiesis in Participants Treated with Mitapivat Agios to Host Live and Webcast Investor Event on Dec. 11, 2023, at 7:00 a.m. Pacific Time CAMBRIDGE, Mass., Dec. 09, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals,...Read more
Oral presentation to include data on 47 patients through five years of follow-up (median 35.5 months, range 0.3-61 months) Endpoints of sVOE-CR and VOE-CR achieved in 94% (32/34) and 88% (30/34) of evaluable patients respectively 100% of adolescents (10/10) experienced complete resolution of VOEs and sVOEs, providing evidence of potential benefit for this population Impact on hemolysis markers and health-related quality of life...Read more
First-ever approval of a CRISPR-based gene-editing therapy in the U.S. Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment Multiple authorized treatment centers activated BOSTON & ZUG, Switzerland / Dec 08, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S....Read more
First-ever approval of a CRISPR-based gene-editing therapy in the U.S. Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment Multiple authorized treatment centers activated BOSTON & ZUG, Switzerland / Dec 08, 2023 / CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S....Read more
Outcomes-based contract offerings available to both commercial payers and Medicaid LYFGENIA will be available through bluebird’s established national network of Qualified Treatment Centers beginning in Q1 2024 “my bluebird support” patient services program will provide personalized support for patients and their families throughout their treatment journey Price of LYFGENIA reflects its value as a potentially curative gene therapy for...Read more
Severe vaso-occlusive events were eliminated for 94% (30/32) of evaluable patients and all VOEs were eliminated for 88% (28/32) of evaluable patients between 6 and 18 months post-infusion LYFGENIA is the most deeply studied gene therapy for sickle cell disease with the most patients treated and longest follow-up Management to host conference call today, December 8, 2023, at 4:15 p.m. ET SOMERVILLE, Mass. / Dec 08, 2023 / Business...Read more
Silver Spring, MD / December 8, 2023 / Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative...Read more
Recursion Pharmaceuticals is a clinical stage TechBio company leading the space by decoding biology to industrialize drug discovery. Enabling its mission is the Recursion OS, a platform built across diverse technologies that continuously expands one of the world’s largest....
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