Rare diseases
The company discontinued development last month of its most mature asset, RLYB212, following disappointing mid-stage pharmacokinetic findings in a rare bleeding disorder.
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
Vertex has recorded some 25,000 prescriptions for Journavx since its January approval and is in the process of getting big PBMs to cover the non-opioid pain drug.
At the heart of the acquisition is Regulus’ farabursen, an miRNA-targeting oligonucleotide in early-stage development for rare autosomal dominant polycystic kidney disease.
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
In an interview with former Fox News journalist Megyn Kelly, FDA Commissioner Marty Makary introduced a new mechanism-driven pathway that could be leveraged by rare disease therapies while saying that autism could potentially be driven by certain environmental factors.
The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a rare disease that affects the entire body and produces a wide range of symptoms.
Lined up for the FDA in the coming weeks are a cell-based gene therapy for a rare skin disease and two product expansions for Regeneron, one with partner Sanofi.
The company is dropping its former lead molecule in favor of another antibody, RLYB116, which is being developed for a variety of rare autoimmune disorders.
Stifel analysts said that Lexeo’s data showing reduced size and thickness of the heart’s left ventricle are “supportive of a drug effect” for the company’s gene therapy in Friedreich’s ataxia cardiomyopathy.
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