Third Quarter 2024 Financial Results: As of September 30, 2024, the Company had $109.0 million in cash, money market fund, time deposits and U.S treasury bills.

Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1. Tinlarebant has been granted Fast Track Designation ...

This Orphan Drug Designation for ATA-200 was granted a few weeks after ATA-200 received Rare Pediatric Disease Designation by the FDA. LGMD-2C/R5 is a rare genetic disease caused by mutations in the ...

An international team of researchers has provided a genetic diagnosis for 30 individuals whose condition was undiagnosed for years despite extensive ...

(NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced that it has ...

Data from the pivotal Phase 2b ReNeu trial demonstrate that patients with NF1-PN achieved deep responses and improvements in ...