The NDA is supported by data from a single-arm, multi-center, open-label, Phase II registration study in China that completed enrollment of 87 patients in March.

Patients submitted for consideration for treatment with GS010/Lumevoq must meet specific eligibility criteria, including the length of time since the onset of their vision loss.

The Recommended Uniform Screening Panel now includes DMD and MLD, which has implications for firms developing treatments for ...

This year, there was a surge of new lung cancer treatments, more options for patients with earlier-stage disease, and growing ...

While autologous therapies are mainstream in blood cancers, experts warn against overhyping allogeneic and in vivo approaches ...

NEW YORK – BioMarin has stopped developing the investigational BMN 349 for treating alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company announced in a regulatory filing this ...

Jacobio will get $100 million upfront in the deal and be entitled to an additional $1.92 billion upon meeting certain milestones related to JAB-23E73.

Endometrial02, researchers want to see whether Enhertu can replace current standard-of-care chemotherapy for patients with HER2-expressing tumors.

NEW YORK – The convergence of gene-editing breakthroughs and new regulatory frameworks signaled a turning point in 2025 for getting gene therapies for rare diseases to patients. It's notoriously ...