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 ...

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

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 ...

(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 ...

Krystal Biotech, Inc. (NASDAQ:KRYS) Q3 2024 Earnings Call Transcript November 4, 2024 Krystal Biotech, Inc. beats earnings expectations. Reported EPS is $0.909, expectations were $0.9. Operator: Thank ...

Additionally, Quince was granted Fast Track designation by the FDA for the ... company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare ...

Innocan Pharma Corporation ("Innocan" or the "Company"), a pioneer in the pharmaceutical and biotechnology industries, announced that it has taken the strategic decision to focus its development ...

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