Fifteen patients with mutations in the GUCY2D gene were treated with increasing doses of ATSN-101. Overall, the subretinal therapy was well tolerated, with most side effects related to the surgery rather than the drug.
The trial also showed patients who received the highest dose of the gene therapy made progress in night vision and overall blindness.
To the researchers’ knowledge, gene therapy ATSN-101 is currently the only gene therapy targeting LCA1.
Becker’s 3rd Annual Fall Chief Pharmacy Officer Summit is the top tier event that the people leading the future of pharmacy convene. This 3-day event is built specifically for Chief Pharmacy Officers and senior pharmacy leaders. The sessions examine the workforce, financial and clinical priorities shaping pharmacy today. Becker’s is where candid discussions flourish and new ideas thrive. Becker's 3rd Annual Fall Chief Pharmacy Officer Summit
