GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported ...

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

Entering 2025 having demonstrated rapid translation of genetic insights to positive clinical data; advancing clinically validated and multi-modal pipeline with the potential to treat well over 100 mil ...

Entering 2025 having demonstrated rapid translation of genetic insights to positive clinical data; advancing clinically validated and multi-modal ...

Advancing INLIGHT clinical study with novel, long-acting, muscle-sparing approach for obesity using Wave’s best-in-class GalNAc-siRNA ...

IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new ...

Equities researchers at Zacks Research cut their Q1 2025 earnings estimates for shares of Sarepta Therapeutics in a research ...

Wave Life Sciences shows promising data in genetic mutation treatments, with GSK collaborations and strong financial footing.

UHealth and Holtz Children’s Hospital physicians are exploring a new gene therapy called ELEVIDYS to treat older Duchenne muscular dystrophy (DMD) patients. The therapy delivers a functional ...

Revidia Therapeutics Inc., a cardiac regenerative medicine company developing first-in-class small molecule drug therapies for heart injury, has announced that it has expanded its leadership team in ...

Capricor's biologics license application for deramiocel (CAP-1002) is draws largely on the Phase 2 HOPE-2 trial and its ...