CAMBRIDGE, Mass., Dec. 18, 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 the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease. Earlier,...Read more
Poster presentation at ASH on Monday, December 9 at 6:00 p.m. PT / 9:00 p.m. ET CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading gene editing company, will present updated safety and efficacy data in 28 patients living with severe sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. The...Read more
Data from long-term follow-up of patients in clinical trials further demonstrate durability of the transformative benefits of CASGEVY™ Safety profile consistent with busulfan conditioning and autologous hematopoietic stem cell transplant Vertex provides update on progress in bringing CASGEVY to patients BOSTON / Dec 08, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for...Read more
Stable production of anti-sickling adult hemoglobin (HBAT87Q) and elimination or significant reduction of vaso-occlusive events sustained through last follow-up for all evaluable patients (n=38), underscoring potentially transformative and durable clinical impact Results from first and only focused analysis of patients with sickle cell disease with a history of overt or silent stroke (n=27) show no stroke recurrence through 9 years of...Read more
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
Compass Therapeutics is a clinical-stage, oncology-focused biopharmaceutical company developing proprietary antibody-based therapeutics to treat multiple human diseases. The company's scientific focus is on the relationship between angiogenesis, the immune system, and tumor growth...
CLICK TO LEARN MORE