Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
Merck Buys Exclusive Rights to Develop Hengrui Pharma's Lp(a) Inhibitor Outside of China
Merck will pay the Chinese pharma company $200 million upfront and up to $1.77 billion in development, regulatory, and commercial milestone payments.
Ontario Brain Institute, EpiSign Partner on Rare Genetic Disorder Diagnosis
OBI's Center for Analytics and EpiSign will refine and validate EpiSign's machine-learning algorithm that analyzes a person's epigenome to detect rare genetic disorders.
Verve Therapeutics to Kick off Base-Editing Cardiovascular Drug Phase I Trial in the US
The firm's Heart-2 study of VERVE-102, which is designed to inactivate the PCSK9 gene, is already underway in Canada and the UK.
Alnylam's RNAi TTR Silencing Drug Amvuttra FDA Approved for Transthyretin Amyloidosis Cardiomyopathy
The drug was already sold for TTR amyloidosis with polyneuropathy, and now is available to patients with hereditary or wild-type ATTR-CM.
Novartis to Seek Approval for Intrathecal SMA Gene Therapy in Children, Young Adults in Early 2025
An intravenous form of the gene therapy is already approved and sold as Zolgensma in the US for patients less than 2 years of age.