Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
FDA OKs Krystal Biotech to Start Clinical Trials for AATD Gene Therapy
KB408 is formulated to be inhaled and designed to deliver two copies of the SERPINA1 gene.
RNA Editing Firm Airna Launches With Plans for AATD Candidate
The company's first area of focus will be a therapeutic program to treat the inherited genetic disease, which is caused by variants in the SERPINA1 gene.
CHMP Recommends Label Extension for Vertex Pharmaceuticals' Cystic Fibrosis Drug
Earlier this year, the drug garnered approval for the same label extension from the FDA, making it available for patients as young as 2 years old.
Canadian Clinic's Struggles Highlight Challenge of Establishing Psychiatric Genetic Counseling
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Though the specialty has had a slow start, a growing body of research on genetics and mental illness makes genetic counselors optimistic about its future.
Rocket Pharma Solidifies Danon Disease Gene Therapy Study Design, Prices Public Stock Offering
The company has settled on a study design for RP-A501 that may allow it to seek accelerated approval and expects to net $175 million through its offering.