Rare diseases
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.
In the coming two weeks, the FDA is expected to announce three big decisions, including one for a dry eye disease therapy.
Soleno’s Vykat XR is the first drug approved for the rare disease that directly targets its hallmark symptom.
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
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