Key Webinar Highlights from Physician Expert Discussing Gene Therapies for Ocular Conditions

We’ve gathered highlights from the Emerging Therapy Solutions® (ETS) webinar hosted January 6th with Dr. Cagri Besirli, MD, PhD from the University of Michigan Kellogg Eye Center on the future of gene therapy for ocular conditions.

• Genetic testing and counseling are now standards of care in the evaluation of people with inherited retinal disease (IRD). IRDs are a group of ocular conditions caused by genetic mutations. To date, over 280 different genes with varying mechanisms related to visual dysfunction have been identified. The collective estimated incidence for IRDs is one per 2,000 people.

• There is currently one United States Food and Drug Administration (FDA) approved gene therapy on the US market for a type of IRD related to the genetic mutation of a gene called RPE65. This therapy, Luxturna® (voretigene neparvovec) from Spark Therapeutics, was approved in 2017.

• The real-world experience of Luxturna in clinical practice has been found to mirror the pivotal clinical trial results. The treatment is well tolerated and has led to improvements in vision and retinal sensitivity.

• Luxturna is offered only at select treatment centers that have the expertise to administer the gene therapy. Contact ETS to see their listing.

• There are numerous active clinical trials utilizing gene therapy for other forms of inherited retinal disease including for X-linked retinitis pigmentosa and achromatopsia. Preliminary data are promising.

• Gene therapy is bringing new hope to people with inherited retinal diseases and the pipeline of new gene therapies for this group of conditions is strong.

Emerging Therapy Solutions® helps healthcare payers – including reinsurance and stop-loss payers, health plans and self-funded employers – manage the risks associated with high-cost therapies for rare and complex conditions. We offer a comprehensive suite of services for solid organ and bone marrow transplants, cell and gene therapies, and other highly specialized therapies.

To learn more about emerging therapies including gene therapies, please contact an ETS Account Manager at info@emergingtherapies.com or 877-445-4822.