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Monday, September 15, 2025

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Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial for the Treatment of Rett Syndrome

Taysha Gene Therapies, a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene […]

Synthego Opens State-of-the-Art GMP Guide RNA Manufacturing Facility to Enable CRISPR Genomic Medicines

Synthego, a leading provider of genome engineering solutions, announced today the opening of its state-of-the-art […]

ElevateBio Announces $401 Million Series D Financing to Further Accelerate Growth

ElevateBio, a technology-driven company focused on powering the creation of life-transforming cell and gene therapies, […]

Touchlight Completes Expansion, Creating One of the World’s Largest DNA Manufacturing Facilities in London

Touchlight, a company pioneering enzymatic DNA production, a critical material in many new genetic medicine […]

Life Biosciences Expands Team with the Appointment of Ming Yang, Ph.D., as SVP of Research and Development

Life Biosciences (“Life Bio”), a biotechnology company advancing innovative cellular rejuvenation platforms to reverse diseases […]

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Rocket Pharmaceuticals, a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic […]

Sangamo Therapeutics Receives U.S. FDA Fast Track Designation for Isaralgagene Civaparvovec for the Treatment of Fabry Disease

Sangamo Therapeutics, a genomic medicine company, today announced that the United States Food and Drug […]

Tune Therapeutics Unveils Breakthrough Data Showing Stable and Durable Epigenetic Regulation in Non-Human Primates

Last week, at the 26th Annual Meeting of the American Society of Gene & Cell […]

FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa

The U.S. Food and Drug Administration approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based […]

Chroma Medicine Announces Data Demonstrating the Benefits of Epigenetic Editing for Multiplex Gene Regulation

Chroma Medicine, a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today presented data demonstrating […]

CANbridge and UMass Chan Medical School Spinal Muscular Atrophy Gene Therapy (CAN203) Improves Lifespan and Motor Function in Mice

CANbridge Pharmaceuticals, a leading China-based global biopharmaceutical company committed to the research, development and commercialization […]

  1. Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients Read more
  2. Verge Genomics Announces Positive Safety and Tolerability Data from the Phase 1 Clinical Trial of VRG50635, a Potential Best-in-Class Therapeutic for All Forms of ALS Read more
  3. DEM BioPharma Appoints Wendy Young, Ph.D., to Scientific Advisory Board Read more
  4. Confo Therapeutics Enters into Research Collaboration for GPCR-Targeting Antibody Discovery with AbCellera Read more
  5. Vyriad Announces Expansion of T-Cell Lymphoma Trial at Mayo Clinic Read more