gene therapies in ophthalmology competitive landscape

DelveInsight’s, “Gene Therapies In Ophthalmology- Competitive landscape, 2025,” report provides comprehensive insights about  55+ companies and 125+ drugs in Gene Therapies In Ophthalmology Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Gene Therapies in Ophthalmology: Understanding

Gene Therapies in Ophthalmology: Overview

Gene therapy is a medical technique that modifies a person's genes to treat or cure disease. It can work by replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning properly, or introducing a new or modified gene into the body to help treat a disease. Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases. 

 

Gene therapy encompasses three facets: gene silencing through siRNA, shRNA, and miRNA; and gene replacement, where the desired gene is administered directly in the form of plasmids and viral vectors. Additionally, it involves gene editing-based therapy, where mutations are modified using specific nucleases like zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regulatory interspaced short tandem repeats (CRISPR)/CRISPR-associated protein (Cas)-associated nucleases. Transfer of gene is either through transformation where under specific conditions the gene is directly taken up by the bacterial cells, transduction where a bacteriophage is used to transfer the genetic material and lastly transfection that involves forceful delivery of gene using either viral or non-viral vectors. The non-viral transfection methods are subdivided into physical, chemical and biological. The physical methods include electroporation, biolistic, microinjection, laser, elevated temperature, ultrasound and hydrodynamic gene transfer. The chemical methods utilize calcium- phosphate, DAE-dextran, liposomes and nanoparticles for transfection. The biological methods are increasingly using viruses for gene transfer, these viruses could either integrate within the genome of the host cell conferring a stable gene expression, whereas few other non-integrating viruses are episomal and their expression is diluted proportional to the cell division.

 

Gene therapy involves the modification of defective deoxyribonucleic acid (DNA) in recipient cells or tissues to achieve a desired therapeutic effect. Compared to other organs, the eye has a greater potential for gene therapy due to its easy accessibility via injections and surgical interventions, immune-privileged status, presence of tight ocular barriers preventing exposure to other organs, and ready assessment of retinal structure and anatomy by non-invasive techniques to determine response to treatment. Also, retinal dystrophies are usually symmetrical and bilateral, allowing one eye to serve as a control in clinical trials. A major disadvantage is that advanced retinal dystrophies or degenerations are usually irreversible, and successful treatment depends on the presence of live neuronal cells at the time of initiating gene therapy.

 

In gene therapy, a cloned copy of the wild-type gene associated with a specific condition is utilized, along with a promoter that precisely regulates gene expression in the target cells. Additionally, a vector or carrier, typically a virus stripped of its replicative and virulent features, is employed to act as a cargo, delivering the required gene into the host's target cells.

Report Highlights

• In December 2023, MeiraGTx Holdings announced an asset purchase agreement with Janssen Pharmaceuticals, Inc. (J&J), a Johnson & Johnson company, for the remaining interests in bota-vec for the treatment of XLRP, as well as a commercial supply agreement and a technology transfer agreement for bota-vec manufacturing.
• In October 2023, OBiO Technology (Shanghai) Corp., Ltd announced an agreement with Refreshgene Therapeutics (Refreshgene). Under the agreement, OBiO leverages its capabilities of process development, large-scale cGMP commercial production and global business development to provide high-quality CDMO/CMO services and authorized cooperation services for Refreshgene's RRG-001 pipeline, and jointly promote the development of advanced therapies to benefit patients around the world.
• In September 2023, ALSA Ventures announced the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd.
• In September 2023 Kriya Therapeutics Inc. and Everads has entered into an exclusive license, collaboration and supply agreement.  The therapy is an emerging treatment for diseases in ophthalmology, including geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dry AMD) and affects approximately 1 million people in the United States.  
• In July 2023, Astellas Pharma Inc and 4D Molecular Therapeutics, Inc announced that they have signed a license agreement under which Astellas gains rights to utilize the intravitreal retinotropic R100* vector invented by 4DMT for one genetic target implicated in rare monogenic ophthalmic disease(s), with options to add up to two additional targets implicated in rare monogenic ophthalmic diseases after paying additional option exercise fees.
• In June 2023, Adverum Biotechnologies, Inc. announced an agreement granting Ray Therapeutics a non-exclusive, royalty bearing license of Adverum’s proprietary RTX 015 (AAV.7m8 intravitreal (IVT) vector capsid to be used in conjunction with Ray’s optogenetics payload. Under the terms of the agreement, Adverum grants Ray Therapeutics a worldwide, non-exclusive license of AAV.7m8 for the prevention, treatment, diagnosis or amelioration of any ocular disorder utilizing Ray’s optogenetics approach. 
• In January 2023, Viatris Inc. announced that it has closed its acquisitions of Oyster Point Pharma and Famy Life Sciences to establish a new Viatris Eye Care Division. Under the terms of a definitive agreement, Viatris acquired Oyster Point Pharma for approximately $415 million in cash upfront, which includes the $11 per share paid to Oyster Point Pharma stockholders through a tender offer and the repayment of the principal amount of certain debt of Oyster Point Pharma.

Gene Therapies in Ophthalmology: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Spark Therapeutics

Spark Therapeutics is a biotechnology pharmaceutical company that develops gene therapy treatments for debilitating genetic diseases. It was founded in 2013 by Katherine A. High, Jeffrey Marrazzo, Jean Bennett, J. Fraser Wright, Beverly Davidson, and Jennifer Wellman. Spark Therapeutics is a subsidiary of Hoffmann-La Roche, and its headquarters are located in Philadelphia, Pennsylvania. The company's lead gene therapy candidate, for RPE65-related blindness, is currently in Phase III clinical trials with the potential to be the first approved gene therapy in the United States. Spark Therapeutics is committed to discovering, developing, and delivering gene therapies to break barriers for people and families affected by genetic diseases, including blindness, hemophilia, lysosomal storage disorders, and neurodegenerative disease.

 

Product Description: LUXTURNA

LUXTURNA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells, as determined by the treating physician. Mutations in the RPE65 (retinal pigment epithelial 65 kDa protein) gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in impairment of vision. Injection of LUXTURNA into the subretinal space results in the transduction of some retinal pigment epithelial cells with a cDNA encoding normal human RPE65 protein, thus providing the potential to restore the visual cycle.

Gene Therapies in Ophthalmology: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: REGENXBIO

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, and AAV9. REGENXBIO and its third-party NAV Technology Platform licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a ""5x'25"" strategy to progress five AAV therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.

 

Product Description: RGX-314

RGX-314 is a one-time subretinal treatment that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for the formation of new leaky blood vessels and retinal fluid accumulation. RGX-314 is being developed as a potentially one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF.

ATMOSPHERE, the first of two planned pivotal trials for the evaluation of subretinal delivery of RGX-314 in patients with wet AMD, is active and enrolling patients. In addition, the company is enrolling patients in AAVIATE, a Phase II trial for the treatment of wet AMD using suprachoroidal delivery of RGX-314. A Biologics License Application (BLA) is expected to be submitted to the United States Food and Drug Administration (FDA) in 2024 based on two pivotal trials, ASCENT and the ongoing ATMOSPHERE trial. The ASCENT clinical trial conducted in partnership with AbbVie, is expected to enroll patients in the United States and Canada.

 

2. Company Overview: Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 with the goal of saving and restoring the vision of patients with a variety of prevalent and rare retinal diseases. The company's lead clinical candidate is AGTC-501, an adeno-associated virus (AAV) gene therapy for X-linked retinitis pigmentosa (XLRP), which is currently in Phase II clinical trials. Beacon Therapeutics was created through the combination of Applied Genetic Technologies Corporation (AGTC) with two other pre-clinical programs. The company also has two preclinical assets in its portfolio, including a program for dry age-related macular degeneration. Syncona Limited, a life sciences investment firm, has a majority stake in Beacon Therapeutics and led the financing round alongside additional investors, including Oxford Science Enterprises

 

Product Description: AGTC-501

AGTC-501 is a gene therapy program currently in a Phase II/III clinical trial for the treatment of X-Linked Retinitis Pigmentosa (XLRP) that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

 

3. Company Overview: Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases.

 

Product Description: ADVM-022

ADVM-022 is a recombinant, replication-deficient adeno virus-associated (AAV) gene therapy. ADVM-022 utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. 

ADVM-022 (AAV.7m8-aflibercept) is administered as a single, in-office intravitreal (IVT) injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for patients with wet AMD and DME. The therapeutic candidate is in Phase II clinical studies for wet AMD and diabetic retinopathy.

 

4. Company Overview: Exegenesis Bio

Exegenesis Bio is committed to developing and increasing access to gene and cell therapies for patients around the world, with a mission to build a fully integrated global gene and cell therapy company with industry-leading R&D, manufacturing, and commercialization capabilities. The company was founded in 2019 with strong financial backing and has built a broad pipeline of innovative genetic medicines and fully integrated manufacturing capabilities. The management team has extensive leadership experience at leading US and global gene and cell therapy companies across all functional areas. Exegenesis Bio has raised over USD 120 million during the past two years and is now expanding globally with offices in Philadelphia, Boston, China, and Singapore. The company is advancing an early-to-clinical-stage gene therapy pipeline with three initial focused therapeutic areas: CNS, ophthalmology, and liver. The company’s vision is to develop and increase access to gene and cell therapies for patients around the world.

 

Product Description: EXG102-031

EXG102-031 intraocular injection is a novel recombinant adeno-associated virus (rAAV)-based gene therapy expressing a therapeutic fusion protein that is able to bind or neutralize all known subtypes of Vascular Endothelial Growth Factor (VEGF) and Angiopoietin-2 (ANG2), which are known to stimulate abnormal blood vessel formation and vascular leakage in the retina. 

EXG102-031 is currently being evaluated in a Phase I/II trial for the treatment of neovascular age-related macular degeneration. 

 

5. Company Overview: Frontera Therapeutics

Frontera is a leading clinical-stage biotechnology company that seeks to develop the best gene therapy medicines to improve the lives of patients across multiple disease areas. Frontera's APEX Technology & Manufacturing Platform is an innovative adeno-associated virus (AAV) gene expression system. It includes novel and clinically validated AAV vectors, efficient and fully integrated CMC processes, and access to early clinical data, making it possible to develop novel gene therapy products with quicker speed-to-market and lower cost. Frontera’s development pipeline spans not only orphan diseases but also targets large patient markets, including ophthalmology, hematology, cardiovascular, neurology and metabolic diseases. Frontera Therapeutics has offices in the United States and China, allowing it to target global patient populations.

 

Product Description: FT-002

FT-002 is a recombinant adeno-associated virus gene therapy drug intended to treat patients with X-linked retinitis pigmentosa (XLRP) caused by an RPGR gene mutation. There is no treatment for this disease globally. FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. One injection can effectively delay disease progression or restore the patient's visual function. FT-002 is the first AAV gene therapy drug to be tested in XLRP patients in China and is also a potential first-in-class drug. FT 002 is currently being evaluated in a Phase I trial for the treatment of X-Linked Retinitis Pigmentosa.

Further product details are provided in the report……..

Gene Therapies in Ophthalmology Analytical Perspective by DelveInsight

• In-depth Commercial Assessment: Gene Therapies in Ophthalmology Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition – deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

• Gene Therapies in Ophthalmology Competitive Landscape 

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

Gene Therapies in Ophthalmology Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

• Current Treatment Scenario and Emerging Therapies:
• How many companies are developing Gene Therapies in Ophthalmology drugs?
• How many Gene Therapies in Ophthalmology drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Gene Therapies in Ophthalmology?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Gene Therapies in Ophthalmology therapeutics? 
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? 
• What are the clinical studies going on for Gene Therapies in Ophthalmology and their status?
• What are the key designations that have been granted to the emerging and approved drugs?