Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

By Karen Roman Biopharmaceutical company Medera Inc. said it completed patient enrollment for Cohort B of its clinical trial ...

Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 gene therapy The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene ...

Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease ...

About the FALCON Trial FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who have been ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...

Leaders from the world of cell and gene therapy, including molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates, shared their personal stories and policy ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

Avista Therapeutics, a preclinical gene therapy company built on deep clinical, computational and gene therapy expertise, today announced the significant expansion of its gene therapy platform.

A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the ...