Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : APX3330
Therapeutic Area : Ophthalmology
Study Phase : Phase II
Sponsor : Ocuphire Pharma
Deal Size : Undisclosed
Deal Type : Merger
Ocuphire Pharma Announces Acquisition of Opus Genetics
Details : The acquisition creates a leading, clinical-stage company focused on developing gene therapies, including APX3330, an oral small-molecule inhibitor of Ref-1 for non-proliferative diabetic retinopathy.
Brand Name : APX3330
Molecule Type : Small molecule
Upfront Cash : Undisclosed
October 22, 2024
Lead Product(s) : APX3330
Therapeutic Area : Ophthalmology
Highest Development Status : Phase II
Sponsor : Ocuphire Pharma
Deal Size : Undisclosed
Deal Type : Merger
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Opus Genetics Receives FDA Rare Pediatric Disease Designation for OPGx-LCA5
Details : OPGx-LCA5 is an 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.
Brand Name : OPGx-LCA5
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 20, 2024
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : OPGx-RHO
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Foundation Fighting Blindness
Deal Size : $1.7 million
Deal Type : Funding
Opus Genetics Announces $1.7 Million in Funding for Preclinical Programs
Details : The proceeds will be used to conduct a preclinical safety study for OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations (RHO-adRP).
Brand Name : OPGx-RHO
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
June 13, 2024
Lead Product(s) : OPGx-RHO
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Foundation Fighting Blindness
Deal Size : $1.7 million
Deal Type : Funding
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Opus Genetics Completes Dosing in Phase 1/2 Trial of Gene Therapy OPGx-LCA5
Details : OPGx-LCA5 uses an AAV8 vector to deliver a functional LCA5 gene to the retina for patients with Leber congenital amaurosis from LCA5 mutations.
Brand Name : OPGx-LCA5
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
March 26, 2024
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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).
Brand Name : OPGx-LCA5
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
September 07, 2023
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : AAv-based Gene Therapy
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Recipient : IVERIC bio
Deal Size : Undisclosed
Deal Type : Acquisition
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.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : $0.5 million
December 28, 2022
Lead Product(s) : AAv-based Gene Therapy
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Recipient : IVERIC bio
Deal Size : Undisclosed
Deal Type : Acquisition
Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : OPGx-001
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 01, 2022
Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : OPGx-001
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 ...
Brand Name : OPGx-001
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 04, 2022
Lead Product(s) : OPGx-001
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : OPGx-002
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : OPGx-002
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
April 28, 2022
Lead Product(s) : OPGx-002
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : OPGx-001
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : National Resilience
Deal Size : Undisclosed
Deal Type : Collaboration
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 OPG...
Brand Name : OPGx-001
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
April 11, 2022
Lead Product(s) : OPGx-001
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : National Resilience
Deal Size : Undisclosed
Deal Type : Collaboration
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