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OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene (LCA5).
Lead Product(s): OPGx-LCA5
Therapeutic Area: Rare Diseases and Disorders Product Name: OPGx-LCA5
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 26, 2024
Details:
OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).
Lead Product(s): OPGx-LCA5
Therapeutic Area: Rare Diseases and Disorders Product Name: OPGx-LCA5
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 07, 2023
Details:
Opus Genetics' AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies.
Lead Product(s): AAv-based Gene Therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Recipient: IVERIC bio
Deal Size: Undisclosed Upfront Cash: $0.5 million
Deal Type: Acquisition December 28, 2022
Details:
OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5gene to retinal photoreceptors. Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001.
Lead Product(s): AAV8.hLCA5
Therapeutic Area: Rare Diseases and Disorders Product Name: OPGx-001
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 01, 2022
Details:
Preclinical data, including faithful animal and human iPSC models, have demonstrated preservation of visual function when OPGx-001 was administered prior to peak disease severity, indicating the potential for a broad therapeutic window for patients with LCA5.
Lead Product(s): OPGx-001
Therapeutic Area: Genetic Disease Product Name: OPGx-001
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 04, 2022
Details:
Data support continued development of OPGx-001, designed to address mutations in LCA5 gene and OPGx-002, restoring protein expression caused by mutations in RDH12 gene, to address mutations in genes causing forms of Leber congenital amaurosis.
Lead Product(s): OPGx-002
Therapeutic Area: Genetic Disease Product Name: OPGx-002
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 28, 2022
Details:
Resilience will provide process and analytical development, quality control testing, and GMP manufacturing services for IND-enabling toxicology and first-in-human material for Opus’ adeno-associated viral (AAV) vector-based gene therapies including OPGx-001, OPGx-002 and OPGx-003.
Lead Product(s): OPGx-001
Therapeutic Area: Genetic Disease Product Name: OPGx-001
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: National Resilience
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 11, 2022
Details:
Agreement signed by Opus Genetics to license its third preclinical program OPGx-003, to address mutations in the NMNAT1 gene which cause a specific form of Leber congenital amaurosis (LCA) program.
Lead Product(s): OPGx-003
Therapeutic Area: Genetic Disease Product Name: OPGx-003
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Massachusetts Eye and Ear and Harvard Medical School
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement November 11, 2021
Details:
The initial seed funding will allow Opus to advance the preclinical research including the lead program, OPGx-001, which is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein.
Lead Product(s): OPGx-001
Therapeutic Area: Rare Diseases and Disorders Product Name: OPGx-001
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: RD Fund
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Financing September 22, 2021
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