D Molecular Therapeutics is undervalued, with its lead candidate 4D-150 showing potential. Click here to find out why I rate ...

The five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three to six times each week.

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

Ocugen, Inc. (NASDAQ: OCGN) has announced the completion of dosing in the Phase 2 portion of its ArMaDa clinical trial for ...

A 31-year-old Sligo man who was functionally blind has got his sight back, after being treated with a new gene therapy at the ...

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing a ...

Currently, patients are treated with injections of asfotase alfa ... "We believe the next evolution in treating HPP will be a gene therapy in which a single injected dose will provide a lifelong ...

This article examines viral and non-viral vectors in gene therapy, highlighting their mechanisms, advantages, and limitations ...