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Friday, September 26, 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 […]

ObvioHealth Announces Ground-Breaking Digital Therapeutics API to Capture More Accurate Efficacy and Adherence Data in Clinical Trials

ObvioHealth, a pioneering virtual research organization, announces the launch of a proprietary application programming interface […]

Neurophth Completes Patient Enrollment for Phase III clinical trial for the Gene Therapy treatment of LHON

Neurophth Therapeutics announced today that the last patient has been enrolled in the Phase III […]

Pharming announces First Patient Enrolled in Pediatric Clinical Trial of leniolisib

Pharming Group announces that the first patient has been enrolled in its Phase III clinical […]

Actinium Announces Positive Full Data Results From the Pivotal Phase 3 SIERRA Trial in Patients with Active, Relapsed or Refractory Acute Myeloid Leukemia

Actinium Pharmaceuticals, a leader in the development of targeted radiotherapies, today announced positive results for […]

Sarepta Therapeutics Announces Initiation of VOYAGENE, a Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4

Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, today announced that the […]

Maze Therapeutics to Present Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease

Maze Therapeutics, a company translating genetic insights into new precision medicines, has announced that the […]

Alterity Therapeutics Receives Regulatory Authorization in France and Austria to Proceed with ATH434 Phase 2 Clinical Trial

Alterity Therapeutics, a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today […]

Jasper Therapeutics Announces Positive Follow-up Clinical Data from Investigator-Sponsored Study of Briquilimab Conditioning in Sickle Cell Disease Patients

Jasper Therapeutics, a biotechnology company focused on developing novel antibody therapies targeting c-Kit (CD117) to […]

Silver Creek Presents ARPEGGIO, a Phase 2 Clinical Trial Evaluating Scp776, a First-in-Class Cell Rescue Drug, for the Treatment of Acute Ischemic Stroke

Silver Creek, a biotechnology company headquartered in San Francisco, presented the ARPEGGIO study at the […]

First Patient in the US Dosed in a Global Multicentre Phase 3 Clinical Study of Bevacizumab for treatment of Ophthalmic Diseases

Essex Bio-Technology today announced that the first patient in the United States (US) was dosed […]

  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