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
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
Study’s Primary Efficacy Endpoint Achieved, with 46.2% of Patients in the 50 mg BID Mitapivat Arm and 50.0% of Patients in the 100 mg BID Mitapivat Arm Achieving a Hemoglobin Response, Compared to 3.7% of Patients in the Placebo Arm A Trend in Sickle Cell Pain Crisis Reduction was Observed in Both Mitapivat Dose Arms Compared to Placebo Company Expects to Enroll First Patient in Phase 3 Portion of Study in Q4 2023, Report Phase 3 Data in 2025 and Potentially... Read more
BLA submission based on data from the largest and most mature clinical development program for any gene therapy in sickle cell disease PDUFA date set for December 20, 2023 SOMERVILLE, Mass. / Jun 21, 2023 / Business Wire / bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review. Lovo-cel is a potentially transformative... Read more
Both trials met the primary and key secondary endpoints at the pre-specified interim analysis Data continue to demonstrate transformative and durable benefit Safety profile consistent with busulfan conditioning and autologous hematopoietic stem cell transplant BOSTON & ZUG, Switzerland / Jun 09, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that both pivotal trials for exagamglogene... Read more
First CRISPR gene-editing filings to be accepted for review by FDA FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) PDUFA target action date of December 8, 2023, for SCD and March 30, 2024, for TDT BOSTON & ZUG, Switzerland / Jun 08, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food... Read more
CAMBRIDGE, Mass., June 06, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that it will host a live webinar on Monday, June 12, at 8:00 a.m. ET to present clinical data from the RUBY trial of EDIT-301 for the treatment of severe sickle cell disease. The Company will also present initial clinical data from the EDITHAL trial of EDIT-301 for the treatment of transfusion-dependent beta thalas... Read more
CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that a scientific abstract detailing safety and efficacy clinical data from the Phase 1/2 RUBY trial of EDIT-301 in patients with severe sickle cell disease has been accepted for an oral presentation at the European Hematology Association (EHA) Hybrid Congress being held June 8-11, 2023, in Frankfurt, Germany, and via live... Read more
CAMBRIDGE, Mass., April 27, 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 Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease. The FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare Pediatric Disease designation to EDIT-301... Read more
BLA submission based on data from the largest and most mature clinical development program for any gene therapy in sickle cell disease SOMERVILLE, Mass. / Apr 24, 2023 / Business Wire / bluebird bio, Inc. (Nasdaq: BLUE) today announced the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history... Read more
EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023 BOSTON & ZUG, Switzerland / Apr 03, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel... Read more
Clinical trial part of long-term growth strategy for motixafortide across multiple potential therapeutic areas TEL AVIV, Israel, March 6, 2023 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, today announced a collaboration with Washington University School of Medicine in St. Louis to advance a Phase 1 clinical trial that will evaluate the safety and feasibility of the Company's... Read more
CAMBRIDGE, Mass., Feb. 24, 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 on February 23, 2023, the U.S. Food and Drug Administration (FDA) verbally informed the company that it has issued a full clinical hold regarding the Investigational New Drug (IND) application for FTX-6058 for the potential treatment... Read more
First two sickle cell disease participants have achieved 100% donor myeloid chimerism through 100 days follow-up Third sickle cell disease participant has now achieved 100% donor myeloid chimerism through 30 days follow-up All three participants have increased their hemoglobin at last follow-up relative to baseline REDWOOD CITY, Calif., Feb. 16, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on developing... Read more
REDWOOD CITY, Calif., Jan. 10, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (“Jasper” or the “Company”), a biotechnology company developing novel antibody therapies and stem cell transplant conditioning agents targeting c-Kit, today announced, as part of an overall portfolio prioritization, that the Company will focus on the development of its lead product candidate, briquilimab (formerly known as JSP191), in chronic diseases and stem cell transplant... Read more
Graphite Bio, Inc. (Nasdaq: GRPH) today announced it is voluntarily pausing the Phase 1/2 CEDAR study of nulabeglogene autogedtemcel (nula-cel) for sickle cell disease (SCD) due to a serious adverse event in the first patient dosed with nula-cel, and the company’s conclusion that the event is likely related to study treatment. As a result, the company will not meet its guidance for initial proof-of-concept data in mid-2023. The decision by Graphite Bio to voluntarily pause... Read more
All three sickle cell disease participants treated with briquilimab successfully engrafted with neutrophil engraftment within 12-16 days First two participants with peripheral blood chimerism at 60 days after allogeneic stem cell transplant achieved 100% donor myeloid chimerism First participant treated has a total hemoglobin level of 13.3 g/dL at five months follow up, increased from 8-9 g/dL at baseline REDWOOD CITY, Calif., Jan. 03, 2023 (GLOBE NEWSWIRE) -- Jasper... Read more
Findings from clinical studies (N=63) through eight years of long-term follow-up (n=3) continue to support ZYNTEGLO as a potentially curative one-time gene therapy for patients with beta-thalassemia who require regular red blood cell (RBC) transfusions through the achievement of durable transfusion independence and normal or near normal total hemoglobin levels Detailed investigations affirm that two cases of anemia in patients with sickle cell disease following treatment... Read more
EDIT-301 was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant Both patients treated with EDIT-301 successfully engrafted and are free of vaso-occlusive events during the follow-up period First patient treated has a total hemoglobin level of 16.4 g/dL and 45.4% fetal hemoglobin five months after treatment with EDIT-301 Company to host a webinar today at 8:00 a.m.... Read more
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to cure serious diseases, today announced that the first patient has been dosed with GPH101, now called nulabeglogene autogedtemcel (nula-cel), in the company’s Phase 1/2 CEDAR trial in people with sickle cell disease (SCD). Nula-cel is an investigational gene editing therapy designed... Read more
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to treat or cure serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to GPH101 for the treatment of sickle cell disease (SCD). GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell... Read more
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