Gene Therapy Restores Vision in First-Ever Trial for Rare, Inherited Blindness
A gene therapy developed by University of Florida scientists, which restored useful vision to most patients with the rare, inherited blindness known as Leber congenital amaurosis type I, or LCA1, in a small trial.
First Cohort Benefits From Atsena Therapeutics XLRS Gene Therapy
UF startup Atsena Therapeutics has announced positive preliminary data for its gene therapy candidate ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
Atsena Therapeutics Announces Positive 12-Month Safety and Efficacy Data From Ongoing Phase I/II Clinical Trial To Treat Leber Congenital Amaurosis
Atsena Therapeutics announced positive results in their 12-month safety and efficacy data from the ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.
Atsena Therapeutics Receives FDA Designation for Treatment of Severe Congenital Eye Disease Using Gene Therapy
Atsena Therapeutics has been granted Regenerative Medicine Advanced Therapy designation from the FDA to continue their research on a cure to Leber congenital amaurosis. Patients with this disease are missing a gene that regulates retinal function and visually-guided behavior, causing blindness at an early age.
Atsena Therapeutics Appoints Linda B. Couto, Ph.D., as Chief Scientific Officer
UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced the appointment of Linda B. Couto, Ph.D., as Chief Scientific Officer (CSO).
Atsena Therapeutics Acquires Exclusive Rights to Gene Therapy for GUCY2D-Associated Leber Congenital Amaurosis
UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced that it has acquired exclusive rights to a gene therapy targeting GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina and is a leading cause of blindness […]
Unraveling Genetic Mutations
Listen to Shannon Boye, Ph.D., an associate professor of pediatrics at the University of Florida, talk about her journey in academia and what led her to try gene therapy in the fight against a mutation causing babies to be born blind. Learn more about Unraveling Genetic Mutations.