Rare diseases
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.
In the Phase III MITIGATE trial, Uplizna cut IgG4-related disease flares by 87% versus placebo.
AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.
Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
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