Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...

Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with Duchenne muscular dystrophy - - Follows recently received ...

Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...

Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with ...

Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...

Candel Therapeutics Inc. shares gained momentum as Wall Street analysts highlighted the potential of its lead cancer drug ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic medicine through UCL Institute of Ophthalmology and Moorfields Eye Hospital, with ...

One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.

Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...