Currently, GenAns Biotech is conducting investigator-initiated trials for several gene therapies, including GA001 for ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

Enrollment Complete in Cohorts 1 and 2 of MyPEAK™-1 Phase 1b/2 Trial of TN-201 for MYBPC3-associated HCM; Positive DSMB ...

Discover insights from MaxCyte's Q2 2025 earnings call, including revised revenue guidance, headwinds impact, and strategies for growth in the cell ...

Cash runway extended into 2028; expected to enable multiple meaningful clinical data read-outs -- APOE program added to ...

Novo Nordisk will advance its acquired ATTR amyloidosis with cardiomyopathy monoclonal antibody (mAb) to Phase III trials.

In this week’s edition of InnovationRx, we look at a biotech entrepreneur treating a rare genetic disease, new tech aimed at ...

Excellos brings its donor-to-dose capabilities—including donor management, apheresis, cell characterization, and GMP ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

At only 6 months, Gerard and Kaitlin Norton’s daughter Madeline was diagnosed with PKAN, a rare neurodegenerative disorder ...