Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

Jan. 27, 2025 — Heart disease remains the leading cause of death in the ... While Most Americans Use a Device to Monitor ... stabilize plaques and ... New Gene Therapy Reverses Heart Failure ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...

Orchard Therapeutics to Receive WORLDSymposium™ New Treatment Award ...

Its DELIVER platform generates capsids that are designed to avoid the liver and target muscle and heart ... gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for use ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.

Low-density lipoproteins (LDL)—commonly known as bad cholesterol—have long been on scientists' radar as a major contributor ...

The FDA has offered positive feedback on possible paths forward for accelerated or traditional approval of FLT201 using data from a single-arm study.