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...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...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...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...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...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...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...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,...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...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...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...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,...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
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...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
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...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...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
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...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
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...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...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...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.,...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,...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...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...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...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....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...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)...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...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...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...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)...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...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...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...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...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...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...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....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...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...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...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...Read more
Immix Biopharma is a clinical-stage biopharmaceutical company pioneering a novel class of CAR-T cell therapies and Tissue-Specific Therapeutics targeting oncology and immuno-dysregulated diseases with >75 patients treated to-date. Our lead cell therapy asset is NXC-201...
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