PDE6B

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

Retrieved on: 
Wednesday, January 24, 2024

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.

Coave Therapeutics to Present at Upcoming Conferences

Retrieved on: 
Thursday, September 28, 2023

Paris, France, September 28, 2023 – Coave Therapeutics (‘Coave’), a genetic medicine company focused on developing life-changing therapies for CNS (Central Nervous System) and eye diseases, announces that CEO Rodolphe Clerval will be presenting to investors and peers at the following Investor and Industry conferences:

Key Points: 
  • Paris, France, September 28, 2023 – Coave Therapeutics (‘Coave’), a genetic medicine company focused on developing life-changing therapies for CNS (Central Nervous System) and eye diseases, announces that CEO Rodolphe Clerval will be presenting to investors and peers at the following Investor and Industry conferences:
    Presenting on October 10 at 13:15 PDT and on panel “Science Slam: Path To Commercialization In Gene Therapy” at 14:00 PDT
    In addition, science and data from Coave’s pipeline programs will be presented at the following Scientific, Medical & Regulatory Conferences:
    Oral presentation by Professor G. Le Meur: “Design of a Phase 1/ 2 Trial to Study the Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients with Retinitis Pigmentosa (RP) Harbouring Mutations in the PDE6B (Phosphodiesterase 6B) Gene Leading to a Defect in PDE6B Expression.”
    Oral Presentation by Dr. M.A.
  • Burlot: “Intrastriatal injection of S0112AAV2-GBA1 is an efficient strategy to treat patients suffering from Parkinson’s disease related to GBA1 mutations.” Presentation on 27 October, 08:30-10:00 CEST (ID# OR65)

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

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."

Coave Therapeutics announces Positive 12-months Data from Ongoing Phase I/II Clinical Trial of CTx-PDE6b in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

Retrieved on: 
Wednesday, May 31, 2023

These positive data support Coave’s preparations for a registrational trial with CTx-PDE6b in this indication.

Key Points: 
  • These positive data support Coave’s preparations for a registrational trial with CTx-PDE6b in this indication.
  • To date, CTx-PDE6b has been administered to 17 patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses.
  • The treatment was administered in the more affected eye while the other eye served as an untreated control.
  • “We are highly encouraged by the safety and efficacy data observed so far in patients who have been treated with CTx-PDE6b.

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

Retrieved on: 
Thursday, December 1, 2022

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.

Coave Therapeutics and Théa Open Innovation sign exclusive licensing, co-development and commercialization agreement for Europe for CTx-PDE6b, a novel gene therapy candidate in retinitis pigmentosa

Retrieved on: 
Thursday, September 16, 2021

The agreement covers Coave's lead investigational gene therapy candidate CTx-PDE6b (formerly HORA-PDE6b) for patients with PDE6b-associated Retinitis Pigmentosa (RP), an inherited retinal disease (IRD).

Key Points: 
  • The agreement covers Coave's lead investigational gene therapy candidate CTx-PDE6b (formerly HORA-PDE6b) for patients with PDE6b-associated Retinitis Pigmentosa (RP), an inherited retinal disease (IRD).
  • "We are delighted to sign this agreement with Tha for CTx-PDE6b, our most advanced candidate, which represents an innovative new gene therapy for people with Retinitis Pigmentosa," said Rodolphe Clerval, CEO of Coave.
  • Under the terms of the agreement, Coave will receive an upfront payment and equity investment of 10 million from Tha.
  • Coave is also eligible to receive up to 65 million for achieving specified clinical development, regulatory and commercial milestones with CTx-PDE6b.

Coave Therapeutics and Théa Open Innovation sign exclusive licensing, co-development and commercialization agreement for Europe for CTx-PDE6b, a novel gene therapy candidate in retinitis pigmentosa

Retrieved on: 
Thursday, September 16, 2021

PARIS, Sept. 16, 2021 /PRNewswire/ -- Coave Therapeutics ('Coave'), a clinical-stage biotechnology company focused on developing life-changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases, has entered into an exclusive licensing, co-development and commercialization agreement with Théa, a leading European speciality Pharma focused on ophthalmology. The agreement covers Coave's lead investigational gene therapy candidate CTx-PDE6b (formerly HORA-PDE6b) for patients with PDE6b-associated Retinitis Pigmentosa (RP), an inherited retinal disease (IRD).

Key Points: 
  • The agreement covers Coave's lead investigational gene therapy candidate CTx-PDE6b (formerly HORA-PDE6b) for patients with PDE6b-associated Retinitis Pigmentosa (RP), an inherited retinal disease (IRD).
  • "We are delighted to sign this agreement with Tha for CTx-PDE6b, our most advanced candidate, which represents an innovative new gene therapy for people with Retinitis Pigmentosa," said Rodolphe Clerval, CEO of Coave.
  • Under the terms of the agreement, Coave will receive an upfront payment and equity investment of 10 million from Tha.
  • Coave is also eligible to receive up to 65 million for achieving specified clinical development, regulatory and commercial milestones with CTx-PDE6b.

Ocugen Provides Business Update and Second Quarter 2020 Financial Results

Retrieved on: 
Friday, August 14, 2020

ET

Key Points: 
  • ET
    MALVERN, Pa., Aug. 14, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing and commercializing transformative therapies to cure blindness diseases, today reported second quarter 2020 financial results along with a general business update.
  • In early August 2020, the FDA granted another ODD for OCU400 for the treatment of phosphodiesterase 6B (PDE6B) mutation-associated RP.
  • Second Quarter 2020 Financial Results:
    In April 2020, Ocugen completed an amendment and exchange of its Series A Warrants for an aggregate of 21.9million shares of Ocugens common stock and non-interest-bearing unsecured notes in the aggregate principal amount of $5.6million.
  • The Company has made prepayments of $3.1million on the unsecured notes from proceeds received under the May 2020 and June 2020 ATMs.