CAMBRIDGE, Mass., Aug. 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). “We are thrilled that the FDA... Read more
Robust and clinically meaningful absolute mean fetal hemoglobin (HbF) induction of 8.6% from baseline at 12 weeks of treatment; 7 of 16 patients achieved absolute HbF levels greater than 20% Evidence of pan-cellular induction of HbF based on an increase in F-cells (red blood cells containing HbF) from a mean of 34% at baseline to 67% at 12 weeks of treatment Meaningful improvements in key markers of hemolysis coupled with a 0.9 g/dL mean increase in total... Read more
European Unitary Patent EP4117446 Validated - Lobe Sciences secures patent protection in 17 EU member states for its DHA and egg yolk-based composition designed to manage Sickle Cell Disease. Covers Altemia(R) Medical Food Formula - The patented composition underpins Altemia(R), a physician-supervised medical food formulated to address long-chain fatty acid deficiencies common in SCD patients. Supports Global Expansion Strategy - Validation strengthens Lobe's... Read more
Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to <40%, and Resolution of Anemia Patients Required a Median of One Mobilization Cycle and Experienced Rapid Neutrophil and Platelet Engraftment Safety Profile Remained Consistent with Busulfan Conditioning, Autologous Hematopoietic Stem Cell Transplantation and Underlying SCD Enrollment... Read more
Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy. Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia. CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating... Read more
CAMBRIDGE, Mass., June 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). “Sickle cell disease is a devasting disorder that affects approximately... Read more
Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101 Beam to Host Investor Webcast on Friday, June 13, 2025, at 4:00 p.m. ET CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present new data from... Read more
Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached in August 2024 LONDON / Jan 31, 2025 / Business Wire / Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autote... Read more
CAMBRIDGE, Mass., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the company will encore data from the BEACON Phase 1/2 clinical trial of BEAM-101 in sickle cell disease in an oral presentation at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and... Read more
First patient enrolled in NIH/NHLBI-sponsored Phase 1 Study of fostamatinib, Rigel's oral SYK inhibitor SOUTH SAN FRANCISCO, Calif., Jan. 22, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced the first patient has been enrolled in a Phase 1 study evaluating the safety and tolerability of escalating doses of fostamatinib, the company's oral spleen tyrosine kinase (SYK) inhibitor, in patients with sickle cell disease (SCD). The study... Read more
Collaboration to leverage Orna's differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ -- Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research collaboration... Read more
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, in November 2020, the U.S. Food and... 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 data will be presented by Dr. Rabi... Read more
NHP Data Showed CD117 Monoclonal Antibody (mAb) Conditioning Successfully Achieved Long-term Engraftment of Base-edited Hematopoietic Stem Cells and Induced Robust Levels of Hemoglobin F mAb Dosing Well Tolerated Without Need for Supportive Care Beam on Track to Initiate Phase 1-enabling Studies by End of 2024 Beam to Host Investor Event on Dec. 8, 2024, at 8 p.m. PT SAN DIEGO, Dec. 08, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology... 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 CASGEVY™ (exagamglogene autotemcel)... 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 follow-up (n=2) SOMERVILLE, Mass.... Read more
All Seven Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to <40%, and Resolution of Anemia Post-BEAM-101 Treatment Initial Safety Profile Consistent with Busulfan Conditioning and Autologous Hematopoietic Stem Cell Transplantation All Seven Patients Dosed Achieved Target Cell Dose with One or Two Mobilization Cycles and Experienced Rapid Neutrophil and Platelet Engraftment Markers of Hemolysis Normalized... Read more
SOMERVILLE, Mass. / Nov 05, 2024 / Business Wire / bluebird bio, Inc. (Nasdaq: BLUE) today announced that new and updated data from its lentiviral vector (LVV) gene addition programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular blood transfusions will be presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place December 7-10,... 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 WALTHAM, Mass., Nov. 5, 2024 /PRNewswire/... 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, 2024, at 8 p.m. PT CAMBRIDGE, Mass.,... 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 safety of mitapivat, an oral, small... 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 (SCD) with recurrent vaso-occlusive... 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 focused on Black/African American... 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 inform subsequent gene therapy conditioning... 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 data for CASGEVY™ (exagamglogene... 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 and autologous hematopoietic stem... 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 optimized process is deployed for 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 the most common inherited diseases... 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 the longest follow-up in the f... Read more
Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review TORONTO, April 1, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission (NDS) for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for the treatment... 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). Approximately 50 percent of... 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 for the treatment of patients... 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 treatment of sickle cell disease... 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 dosed in the Phase 1 clinical trial... 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 Wire / Vertex Pharmaceuticals Incorporated... 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 transfusion-dependent beta thalassemia [TDT])... 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, Inc. (Nasdaq: AGIO), a leader in the... 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 measures further support potential therapeutic... 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. Food and Drug Administration (FDA)... 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. Food and Drug Administration (FDA)... 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 sickle cell disease through... 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 Wire / bluebird bio, Inc. (Nasdaq:... 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 advancement in the field of gene therapy... Read more
First regulatory authorization of a CRISPR-based gene-editing therapy in the world CASGEVY is indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available CASGEVY is indicated for the treatment... Read more
Oral presentation details efficacy, safety and health-related quality of life data in adult and pediatric patients with sickle cell disease treated with lovo-cel in HGB-206 Group C and HGB-210 (n=47) through 5 years of follow-up (n=4) Updated long-term efficacy, safety, quality of life data and iron management outcomes from adult and pediatric patients with transfusion-dependent beta-thalassemia (n=63) treated with beti-cel through 9 years of follow-up (n=1) will be delivered... Read more
Exa-cel PDUFA target action date is December 8, 2023 for severe sickle cell disease (SCD) ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of SCD in people ages 12 and older with recurrent va... Read more
Highest priority programs – BEAM-101 and ESCAPE for sickle cell disease and BEAM-302 for alpha-1 antitrypsin deficiency – expected to provide foundation for meaningful value creation Company to explore partnership opportunities for continued development of select programs Anticipated cost savings, which includes an approximately 20% reduction in workforce, expected to extend the company’s cash runway into 2026 CAMBRIDGE, Mass., Oct. 19, 2023 (GLOBE NEWSWIRE)... Read more
CAMBRIDGE, Mass., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene editing medicine, for the treatment of severe sickle cell disease (SCD). “Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and... Read more
CAMBRIDGE, Mass., Aug. 22, 2023 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Investigational New Drug (IND) application for FTX-6058 for the potential treatment of sickle-cell disease (SCD). “We are pleased with the FDA’s... Read more
SOMERVILLE, Mass. / Aug 16, 2023 / Business Wire / bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has communicated that an advisory committee meeting will not be scheduled for lovotibeglogene autotemcel (lovo-cel). Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) with a proposed indication for patients ages 12 and older who have a history of vaso-occlusive... Read more
