Rare diseases
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.
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
Ionis will receive $280 million upfront and could get up to $660 million in future milestone payments. Ono will take charge of late-stage development as well as regulatory and commercialization activities.
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