
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : OPGx-BEST1
Therapeutic Area : Ophthalmology
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : OPGx-BEST1 is a Cell & Gene Therapy drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Bestrophinopathy.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
September 22, 2025
Lead Product(s) : OPGx-BEST1
Therapeutic Area : Ophthalmology
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : OPGx-BEST1
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Clears IND For Opus Genetics’ Gene Therapy OPGx-BEST1
Details : OPGx-BEST1 is a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD including best disease, or vitelliform macular dystrophy.
Product Name : OPGx-BEST1
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 18, 2025
Lead Product(s) : OPGx-BEST1
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus gets RMAT Designation for OPGx-LCA5 Gene Therapy in Vision Restoration
Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 06, 2025
Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus Shares One-Month Data from Pediatric Gene Therapy OPGx-LCA5 Trial
Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 08, 2025
Lead Product(s) : AAV8.hLCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Phentolamine Mesylate
Therapeutic Area : Ophthalmology
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus Presents Phentolamine Ophthalmic Data at World Cornea Congress IX
Details : Nyxol (phentolamine ophthalmic solution 0.75%) is being evaluated for the treatment in patients with dim light disturbances and decreased mesopic vision.
Product Name : Nyxol
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 21, 2025
Lead Product(s) : Phentolamine Mesylate
Therapeutic Area : Ophthalmology
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Perceptive Advisors
Deal Size : $21.4 million
Deal Type : Public Offering
Opus Genetics Prices Public Offering & Private Placement Over $20M
Details : Opus intends to use the net proceeds to fund clinical development of its lead gene therapy programs, OPGx-LCA5 for the treatment of leber congenital amaurosis.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
March 21, 2025
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Perceptive Advisors
Deal Size : $21.4 million
Deal Type : Public Offering

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Lead Product(s) : Phentolamine Mesylate
Therapeutic Area : Ophthalmology
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus Gets FDA Fast Track for Phentolamine Ophthalmic
Details : Nyxol (phentolamine mesylate) an alpha1/2 adrenergic receptor inhibitor, is being investigated for decreased visual acuity under low light conditions following keratorefractive surgery.
Product Name : Nyxol
Product Type : Miscellaneous
Upfront Cash : Inapplicable
February 26, 2025
Lead Product(s) : Phentolamine Mesylate
Therapeutic Area : Ophthalmology
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus Genetics Announces Updates on OPGx-LCA5 Clinical Program
Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 18, 2025
Lead Product(s) : OPGx-LCA5
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : APX3330
Therapeutic Area : Ophthalmology
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Opus Genetics Receives FDA Agreement for Phase 3 Trial of APX3330 in Diabetic Retinopathy
Details : APX3330 is an oral small-molecule inhibitor of Ref-1, which is currently being evaluated for the treatment of patients with non-proliferative diabetic retinopathy.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 19, 2024
Lead Product(s) : APX3330
Therapeutic Area : Ophthalmology
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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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.
Product Name : Undisclosed
Product Type : Miscellaneous
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
