One Time Injection With Spark’s Gene Therapy LUXTURNA Demonstrated Lasting Visual Improvement

MedicalResearch.com Interview with:

Stephen R. Russell, MD Dina J Schrage Professor of Macular Degeneration Research Service Director, Vitreoretinal Diseases and Surgery Professor of Ophthalmology and Visual Sciences The University of Iowa

Dr. Russell

Stephen R. Russell, MD
Dina J Schrage Professor of Macular Degeneration Research
Service Director, Vitreoretinal Diseases and Surgery
Professor of Ophthalmology and Visual Sciences
The University of Iowa

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: This study examines the efficacy (and safety) of treating children and adults with a form of retinitis pigmentosa known as RPE65-associated Lebers congenital amaurosis, with an adeno-associated viral vector(AAV) delivered RPE65 construct.  Building on successful phase 1/2b trials from multiple centers, the AAV-hRPE65v2 agent now designated as voretigene neparvovec, contains a highly optimized enhancing sequence and promoter.

The main findings were an improvement on a multiple light level mobility test (MLMT) and multiple additional supportive secondary endpoints which included improvements in full-field light sensitivity, Goldmann visual field, and others.

MedicalResearch.com: What should clinicians and patients take away from your report?

Response: The outcomes of this trial demonstrate a strong, statistically and clinically significant improvement with gene therapy in this condition.

  • First, the study represent a milestone of phase 3 completion by a gene therapy study that met its pre-specified primary and several supportive secondary endpoints.
  • Second, the results demonstrate successful improvement of a genetic disease with intervention, rather than a reduction of loss.
  • Third, the safety profile suggests that the adeno-associated vector platform demonstrated little to no inflammation when injected into the subretinal space.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Combined with another recent publication that suggests that up to 75% of known retinal degeneration-causing genes are small enough to fit into an AAV payload, these results support a targeted approach of additional trials for other inherited retinal diseases (IRDs).

Disclosures: My institution has received grants from the sponsor, Spark Therapeutics.  Additionally, I have consulted for Spark.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation:

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Russell, Stephen et al. The Lancet , Volume 0 , Issue 0 ,
DOI: http://dx.doi.org/10.1016/S0140-6736(17)31868-8
 

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

 

 

 

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