BCL11A

Editas Medicine Announces Fourth Quarter and Full Year 2023 Results and Business Updates

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星期三, 二月 28, 2024
Adolescence, Research, Biotechnology, Gene editing, ASH, FDA, Food, File, TDT, BLA, BCL11A, Safety, Society, Collaboration, Vertex Pharmaceuticals, Patent, Editas Medicine, Patient, Cas9, Sickle cell disease, Public expenditure, Poster, Pharmaceutical industry

CAMBRIDGE, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the fourth quarter and full year 2023 and provided business updates.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the fourth quarter and full year 2023 and provided business updates.
  • In 2023, Editas Medicine strengthened and focused its discovery organization to build an in vivo gene editing pipeline.
  • Editas Medicine plans to participate in the following investor events:
    The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m.
  • ET to provide and discuss a corporate update and financial results for the fourth quarter and full year of 2023.

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

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星期二, 一月 2, 2024
SCD, Medical Subject Headings, Marketing, Febrile neutropenia, European Commission, Sickle cell disease, INN, Limited, Hematology, Physician, ATC, EMA, B cell, International, ATMP, HLA, Vomiting, Headache, Thalassemia, Nausea, Tissue, Disease, Civil service commission, HSC, CD34, Cas9, Haematopoiesis, Vertex Pharmaceuticals, Public, Gene, Transplant, Medication package insert, Volatile organic compound, Committee, Patient, Liver disease, TDT, HBF, Mouth ulcer, Cell, Anatomy, Medicine, Hemoglobinopathy, BCL11A, Health, CHMP

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Key Points: 


Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Editas Medicine and Vertex Pharmaceuticals Enter into Non-exclusive License Agreement for Cas9

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星期三, 十二月 13, 2023
Broad Institute, Sickle cell disease, Gene editing, Editas Medicine, Vertex, CRISPR, Rockefeller University, Cas9, Patent, Vertex Pharmaceuticals, BCL11A, Harvard University, Pharmaceutical industry

CAMBRIDGE, Mass., Dec. 13, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the Company and Vertex Pharmaceuticals entered into a license agreement. Under terms of the agreement, Vertex will obtain a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of sickle cell disease and beta thalassemia, including CASGEVY™ (exagamglogene autotemcel). This agreement extends Editas Medicine’s cash runway into 2026.

Key Points: 
  • CAMBRIDGE, Mass., Dec. 13, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the Company and Vertex Pharmaceuticals entered into a license agreement.
  • Under terms of the agreement, Vertex will obtain a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of sickle cell disease and beta thalassemia, including CASGEVY™ (exagamglogene autotemcel).
  • Editas Medicine is the exclusive licensee of certain CRISPR patent estates for making human medicines.
  • The Cas9 gene editing technology provides access to a broad range of genetic mutations, delivering a precise and targeted approach to gene editing medicines.

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

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星期一, 十二月 18, 2023
Medicine, Vomiting, B cell, Anatomy, ATC, Gene, Sickle cell disease, European Commission, Health, International, Transplant, HSC, Headache, INN, Thalassemia, Tissue, Vertex Pharmaceuticals, Liver disease, Disease, Febrile neutropenia, EMA, Civil service commission, Patient, Medication package insert, HBF, Hemoglobinopathy, Limited, CHMP, Mouth ulcer, Marketing, Committee, Advanced Therapy Medicinal Product, HLA, Medical Subject Headings, ATMP, Cas9, Nausea, TDT, BCL11A, Hematology, Volatile organic compound, Public, SCD, Physician, Cell, CD34, Haematopoiesis, Instrumentation, Vaccine, Veterinary medicine, Dentistry, Dietary supplement, Pharmaceutical industry, Medicinal plants, Anemia

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

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Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Beam Therapeutics Reports Pipeline Updates and Second Quarter 2023 Financial Results

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星期二, 八月 8, 2023
Well, Tissue, ESCAPE, Research and development, HBF, Biotechnology, Allele, Gene editing, SCD, Clinical trial, Cancer, SMART Process Acceleration Development Environment, G&A, CD34, BCL11A, AATD, Fetal hemoglobin, CGMP, Patient, Investment, Security (finance), Administration, Cas9, Hematology, Sickle cell disease, Liver, Growth, Hospital, Trial of the century, LNP, LNP Media Group, R, Services General Administration and Coordination Department, Government of Sindh, Treatment, Safety, Pharmaceutical industry, Vaccine, Medical imaging, Television, Beam

CAMBRIDGE, Mass., Aug. 08, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported second quarter 2023 financial results and provided an update on its clinical and pipeline progress.

Key Points: 
  • Beam continues to expect initiation of current good manufacturing practice compliant operations at its North Carolina manufacturing facility in late 2023.
  • Research & Development (R&D) Expenses: R&D expenses were $97.6 million for the second quarter of 2023, compared to $74.6 million for the second quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $24.7 million for the second quarter of 2023, compared to $24.1 million for the second quarter of 2022.
  • Net Loss: Net loss was $82.8 million for the second quarter of 2023, or $1.08 per share, compared to $72.0 million for the second quarter of 2022, or $1.02 per share.

EdiGene Announces Completion of Last Patient Dosing in Phase I Clinical Trial of ET-01, its Investigational Gene-editing Hematopoietic Stem Cell Therapy for Transfusion Dependent β-thalassemia

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星期五, 十一月 18, 2022
Technology, Research, Genetics, Other Technology, Clinical Trials, Health, General Health, Other Science, Science, Trial of the century, NMPA, TDT, Gene editing, Cas9, National Medical Products Administration, BCL11A, CRISPR, Safety, Translation, IND, RNA, Stem cell, Patient, Pharmaceutical industry, Vaccine

The current standard treatment for TDT requires lifelong packed red blood cell transfusions and iron chelation therapy.

Key Points: 
  • The current standard treatment for TDT requires lifelong packed red blood cell transfusions and iron chelation therapy.
  • ET-01 is designed to achieve a functional cure with a single administration of CRISPR/Cas9 gene-modified autologous hematopoietic stem and progenitor cells.
  • ET-01 is an autologous CD34+ hematopoietic stem/progenitor cell with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9.
  • It is the first gene-editing experimental therapy and the first hematopoietic stem cell experimental therapy with IND application approval by China National Medical Products Administration (NMPA).

EdiGene to Present Preliminary Safety and Efficacy Results of an Investigator Initiated Trial for ET-01, its Investigational Gene Editing Hematopoietic Stem Cell Therapy, at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition

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星期四, 十一月 3, 2022
Health, Stem Cells, Genetics, Research, Science, Pharmaceutical, Biotechnology, Patient, Stem cell, RNA, Chinese Academy of Sciences, Cas9, Research, CD34, Society, Institute, Peking Union Medical College, NMPA, TDT, IND, Knowledge, ASH, Regenerative medicine, CEO, National Medical Products Administration, Fetal hemoglobin, Medicine, Chinese Academy of Medical Sciences COVID-19 vaccine, Gene editing, Exposition, CRISPR, Transfusion, BCL11A, Hope, Pharmaceutical industry, Medical device, Vaccine, Hematology

It is reported to be approximately 15,000 to 30,000 patients with -Thalassemia Major (-TM) in China.

Key Points: 
  • It is reported to be approximately 15,000 to 30,000 patients with -Thalassemia Major (-TM) in China.
  • A recent cross-sectional survey initiated by the Institute of Hematology & Blood Diseases Hospital found that only 7% of the samplings are expected to live beyond age 18.
  • The safety profile of ET-01 was consistent with that of autologous hematopoietic stem cell transplantation and myeloablative conditioning.
  • ET-01 is currently in a multi-center Phase I clinical trial in China, with subjects enrollment completed earlier this year.

EdiGene Obtains Worldwide License from Boston Children’s Hospital to Methods and Compositions for Increasing Fetal Hemoglobin Levels to Treat Hemoglobinopathies

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星期三, 三月 16, 2022
Biotechnology, Health, Genetics, Stem Cells, Pharmaceutical, Harvard Medical School, Adolescence, Fetal hemoglobin, Erythropoiesis, National, Hospital, BCL11A, National Medical Products Administration, Technology, Method, U.S. News & World Report, Facebook, Patient, Boston, Center for Drug Evaluation and Research, CDE, Therapy, BCH, News, Center, Hemoglobinopathy, Howard Hughes Medical Institute, National academy, NMPA, National Academy of Medicine, Investigational New Drug, IND, Blog, Gene editing, Research, Lists of diseases, Cancer, CD34, CEO, Social media, RNA, HPFH, Gene expression, Intellectual property, Stem cell, YouTube, Cas9, Pharmaceutical industry, Boston Children’s Hospital, EdiGene, Inc, BOSTON CHILDREN’S HOSPITAL, EDIGENE, INC

Fetal hemoglobin is naturally shut down after birth but can be reactivated by disrupting the BCL11A gene expression during erythropoiesis.

Key Points: 
  • Fetal hemoglobin is naturally shut down after birth but can be reactivated by disrupting the BCL11A gene expression during erythropoiesis.
  • In -thalassemia patients with hereditary persistence of fetal hemoglobin (HPFH), naturally occurring genetic variants lead to higher fetal hemoglobin level and significantly alleviate symptoms, suggesting the potential of this therapeutic approach, said Dong Wei, Ph.D., CEO of EdiGene.
  • Boston Childrens Hospital is ranked the #1 childrens hospital in the nation by U.S. News & World Report and is the primary pediatric teaching affiliate of Harvard Medical School.
  • Founded as a 20-bed hospital for children, Boston Childrens is now a 415-bed comprehensive center for pediatric and adolescent health care.

Sangamo Announces Transition of SAR445136 Sickle Cell Disease Program From Sanofi to Sangamo

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星期四, 一月 6, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, EMA, Clinical trial, Therapy, COVID-19, Safety, Sickle cell disease, Global Research, Development, Program, BCL11A, Gene editing, Investor, Research, Patient, VOC, Stem cell, HBF, U.S. Securities and Exchange Commission, Company, ASH, Annual report, Week, CEO, SCD, FDA, Fast track (FDA), Collaboration, Technology, AES, Fetal hemoglobin, Nasdaq, Hemoglobinopathy, ESE, Anti-transglutaminase antibodies, Pharmaceutical industry, Vaccine, Sangamo Therapeutics, Sanofi, the PRECIZN-1 Study, Sangamo Therapeutics, THE PRECIZN-1 STUDY, SANGAMO THERAPEUTICS

The Companies are collaborating on an orderly transition, while Sangamo explores options to advance the program, including seeking a new collaboration partner.

Key Points: 
  • The Companies are collaborating on an orderly transition, while Sangamo explores options to advance the program, including seeking a new collaboration partner.
  • Sanofi has elected to transition the SCD program to Sangamo following a recent change in Sanofis cell therapy strategy.
  • Sangamo has been a good partner and this decision is not a reflection on the potential of the SAR445136 program.
  • We remain committed to progressing this program and believe SAR445136 has the potential to relieve people living with sickle cell disease of some of their most challenging symptoms.

Sangamo Therapeutics Announces Updated Preliminary SAR445136 Phase 1/2 Proof-of-Concept Data Showing Tolerability and Sustained Effects in Sickle Cell Disease

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星期日, 十二月 12, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Therapy, HBF, U.S. Securities and Exchange Commission, Standard of care, Fetal hemoglobin, FDA, BCL11A, Investor, AES, Clinical trial, Fast track (FDA), Anti-transglutaminase antibodies, Nasdaq, COVID-19, EMA, Annual report, Week, Sickle cell disease, Risk, VOC, Research, Gene editing, Safety, Patient, ESE, Program, Stem cell, SCD, Degenerative disease, Technology, Society, Pharmaceutical industry, Vaccine, Sanofi, Sangamo Therapeutics, the PRECIZN-1 study, Sangamo Therapeutics, THE PRECIZN-1 STUDY, SANGAMO THERAPEUTICS

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary proof-of-concept clinical data from the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development with Sanofi for the treatment of sickle cell disease (SCD).

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary proof-of-concept clinical data from the Phase 1/2 PRECIZN-1 study of SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development with Sanofi for the treatment of sickle cell disease (SCD).
  • We believe this is an important demonstration of our versatile zinc finger platform translating meaningfully into the clinic.
  • Total hemoglobin stabilized by Week 26 after treatment with SAR445136 in all four patients.
  • PRECIZN-1 is an ongoing first-in-human, open label, single arm, multi-site Phase 1/2 study in up to eight patients with SCD evaluating the safety and tolerability of cell therapy candidate SAR445136.