Durham-based Atsena Therapeutics has raised $150 million in an oversubscribed Series C financing led by Bain Capital to advance gene therapies for inherited blindness conditions.
Alkeus Pharmaceuticals will present results from clinical studies of oral gildeuretinol for geographic atrophy secondary to age-related macular degeneration (SAGA) and Stargardt disease (TEASE).
Atsena Therapeutics' gene therapy ATSN-201 has received both Orphan Drug and Rare Pediatric Disease designations from the FDA for X-linked retinoschisis, a rare genetic disorder causing vision loss primarily in males.
Atsena Therapeutics' ATSN-201 gene therapy showed positive safety and early efficacy in the first cohort of XLRS patients, with two of three patients experiencing extensive schisis resolution beginning at 8 weeks post-treatment.
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