Krystal Biotech Touts Positive KB407 CF Gene Therapy Data, Eyes Repeat Dosing and Registrational Path

Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...
Seeking Alpha

SELLAS Announces Positive Data from Preclinical Studies Indicating ASXL1 Mutations as Predictor of Response to SLS009 in Solid Cancers

- Preselection Method for Cancers Responding to SLS009: High Efficacy of SLS009 Observed in 67% of ASXL1 Mutated Solid Cancers vs 0% in Non-ASXL1 Mutated Cancers – - ASXL1 Mutations Predictably ...
eLife

Separating selection from mutation in antibody language models

This important study introduces a new biology-informed strategy for deep learning models aiming to predict mutational effects in antibody sequences. It provides solid evidence that separating ...
Seeking Alpha

Ocugen, Inc. Announces Positive 2-Year Data Across Multiple Mutations from Phase 1/2 Clinical Trial of OCU400 —A Novel Modifier Gene Therapy for Retinitis Pigmentosa

MALVERN, Pa., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and ...
News Medical

How Do Genetic Mutations Cause Disease?

Genetic disorders can occur due to mutations in one gene (monogenic), multiple genes (multifactorial inheritance), and mutation in one or more chromosomes. Point mutations are where one nucleotide in ...