India has over 180 children with Hunter Syndrome, but many people are not even aware of this genetic disorder which mostly ...

CGT Global, a leading biotechnology company driving the development and delivery of innovative cell and gene therapies, today ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

There's a fresh push to edit the genes of human embryos to prevent diseases and enhance characteristics that parents value.

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

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

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

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 ...

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