The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

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.

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

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

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

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

The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

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 ...

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approvalOn track to dose first patient in Phase 3 trial in ...

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...

as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene replacement and CAR-T cell therapies, we discuss the main advances, ...