PDE6A

SparingVision Reaches Final Dose Escalation Step in PRODYGY Trial with SPVN06 for retinitis pigmentosa

Retrieved on: 
Wednesday, January 24, 2024
Data monitoring committee, Patient, RP, DSMB, PDE6A, Geographic atrophy, Retina, Mutation, IND, Epistasis, Disease, Paratriathlon, Safety, PDE6B, Investigational New Drug, KOL, RHO, Retinitis pigmentosa, Part Two, RCD, Pharmaceutical industry

The trial has now progressed to the final dose cohort of the dose-escalation phase (Part 1).

Key Points: 
  • The trial has now progressed to the final dose cohort of the dose-escalation phase (Part 1).
  • SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background.
  • SparingVision is initially focusing on mid-stage RP, one of the leading inherited causes of blindness globally.
  • This progress of the PRODYGY trial to the third cohort at the highest dose of SPVN06 follows an earlier positive recommendation from the DSMB to dose the second cohort at the medium dose in August 2023.

SparingVision Reports Positive Initial Safety Data from the first cohort treated in its PRODYGY Phase I/II Gene Therapy Trial

Retrieved on: 
Wednesday, August 30, 2023
Retina, PDE6B, Moran Eye Center, University, DSMB, AMD, Safety, Retinitis pigmentosa, Hope, Mutation, Research, Paratriathlon, Epistasis, Genomics, AAV, RP, PDE6A, Toys "R" Us, Inc. v. Step Two, S.A, Data monitoring committee, Children's National Hospital, University of Pittsburgh Medical Center, Oxidative stress, Patient, Ophthalmology, RCD, Cone, UPMC, RHO, Phase, Disease, Pharmaceutical industry

SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background.

Key Points: 
  • SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background.
  • Safety data collected on the first three patients (cohort 1) treated with a low dose showed that SPVN06 has been well tolerated with a favorable safety profile.
  • Following review of the data, the Data Safety Monitoring Board (DSMB) concluded that it is safe to initiate the second cohort at a medium dose.
  • We are eagerly awaiting the results of this Phase I/II trial and the potential for this therapy to slow or stop the progression of RP."

SparingVision’s lead asset SPVN06 clears IND application in the US for the treatment of retinitis pigmentosa

Retrieved on: 
Thursday, December 1, 2022
Science, FIH, Professor, DSMB, RCD, Natural history, ANSM, Food, University, DNA, Epistasis, Gene, IND, University of Pittsburgh Medical Center, Safety, PDE6B, Toys "R" Us, Inc. v. Step Two, S.A, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PDE6A, Retina, Retinitis pigmentosa, Disease, RHO, CTA, IRD, AMD, Research, Quality of life, Severe cognitive impairment, Data monitoring committee, Oxidative stress, AAV, FDA, IRDS, Pharmaceutical industry, Vaccine, RP

SparingVision has also submitted a clinical trial authorisation (CTA) application to the French regulator (ANSM), which is currently under review.

Key Points: 
  • SparingVision has also submitted a clinical trial authorisation (CTA) application to the French regulator (ANSM), which is currently under review.
  • First safety data are anticipated in 2023 and the primary endpoint is expected to be reached in 2025.
  • With over 80 genes involved in RP, each with numerous causative mutations, we need to go beyond the gene-by-gene treatment approach.
  • SPVN06 has the potential to become the universal therapeutic solution that patients need, and we are excited for the next phase of development.