Gene Therapies In Ophthalmology Clinical Trial Pipeline Appears Robust With 55+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

The gene therapies in ophthalmology pipeline and clinical trial analysis report deliver important insights on ongoing research, clinical strategies, upcoming therapeutics, and commercial analysis.

New York, USA, Feb. 19, 2024 (GLOBE NEWSWIRE) — Gene Therapies In Ophthalmology Clinical Trial Pipeline Appears Robust With 55+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight 

The gene therapies in ophthalmology pipeline and clinical trial analysis report deliver important insights on ongoing research, clinical strategies, upcoming therapeutics, and commercial analysis. 

DelveInsight’s Gene Therapies In Ophthalmology Competitive Landscape – 2024 report provides comprehensive global coverage of available, marketed, and pipeline gene therapies in ophthalmology in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space, company assessment, comparative assessment, and future growth potential of the gene therapies in ophthalmology competitive domain.

Key Takeaways from the Gene Therapies In Ophthalmology Competitive Landscape Report

  • Over 55+ companies are evaluating 125+ gene therapies in ophthalmology in various stages of development, and their anticipated acceptance in the ophthalmology market would significantly increase market revenue. 
  • Key gene therapies in ophthalmology companies such as Spark Therapeutics, Regenxbio, Beacon Therapeutics, Adverum Biotechnologies, Exegenesis Bio, Frontera Therapeutics, HuidaGene Therapeutics, Nanjing IASO Biotherapeutics, GenSight Biologics, Sylentis, Neurophth Therapeutics, Johnson & Johnson Innovative Medicine, Nanoscope Therapeutics, Eyevensys, Atsena Therapeutics Inc., Coave Therapeutics, OCUGEN, INC, Visgenx, Amarna Therapeutics, Ikarovec, Homology Medicines, Ray Therapeutics, Shanghai Refreshgene Technology Co., Ltd., Complement Therapeutics, Abeona Therapeutics, and others are evaluating new gene therapies in ophthalmology to improve the treatment landscape.
  • Promising gene therapies in ophthalmology such as LUXTURNA, RGX-314, AGTC-501, ADVM-022, EXG102-031, FT 002, HG202, CT103A, GS010, SYL1001, NFS-01, JNJ 81201887, MCO-010, EYS 606, ATSN-201, CTx PDE6B, OCU 400, VGX 0111, AMA 004, IKC159V, Eye disorder Research project, RTx-021, RRG001, CTx 001, ABO-505, and others are under different phases of gene therapies in ophthalmology clinical trials.
  • In December 2023, MeiraGTx Holdings announced an asset purchase agreement with Janssen Pharmaceuticals, Inc., 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. 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 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 had 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 USD 415 million in cash up front, which includes the USD 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.

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Gene Therapies In Ophthalmology Overview

Gene therapies in ophthalmology represent a revolutionary approach to treating a variety of genetic and inherited eye disorders. This innovative field harnesses the potential of genetic manipulation to address the root causes of these conditions, offering the promise of long-lasting and potentially curative treatments. One of the most notable applications of gene therapy in ophthalmology is for the treatment of inherited retinal diseases (IRDs). These conditions, such as retinitis pigmentosa and Leber congenital amaurosis, are often caused by mutations in specific genes that play crucial roles in the function of the retina. Gene therapies aim to introduce functional copies of these genes into the affected cells, restoring their normal function and halting the progression of vision loss.

Adeno-associated viruses (AAVs) are commonly used as vectors for delivering therapeutic genes into target cells in the eye. These viruses are modified to carry the therapeutic gene without causing disease, making them a safe and effective delivery system. Once delivered, the therapeutic gene integrates into the patient’s cells, allowing for the production of the missing or malfunctioning protein.

Luxturna, an FDA-approved gene therapy, exemplifies the success of this approach. It targets a specific genetic mutation causing Leber congenital amaurosis and has demonstrated significant improvement in vision among treated individuals. Luxturna’s success has paved the way for the development of additional gene therapies targeting various genetic mutations associated with retinal disorders.

Find out more about gene therapies in ophthalmology @ Gene Therapies In Ophthalmology Treatment

Gene Therapies In Ophthalmology Pipeline Analysis: Drug Profile

RGX-314: REGENXBIO

RGX-314 represents a singular subretinal therapy administered just once, utilizing the NAV AAV8 vector carrying a gene responsible for a monoclonal antibody fragment. The resultant protein aims to counteract the activity of vascular endothelial growth factor (VEGF), thereby altering the process of forming new permeable blood vessels and the accumulation of fluid in the retina. RGX-314 is in the developmental stage as a potential single-dose remedy for wet AMD, diabetic retinopathy, and various other chronic retinal conditions typically treated with anti-VEGF interventions.

The initial of the two planned pivotal trials assessing subretinal delivery of RGX-314 in patients with wet AMD, known as ATMOSPHERE, is currently active and accepting patient enrollments. Additionally, the company is actively enrolling patients for AAVIATE, a Phase II trial focused on treating wet AMD through suprachoroidal delivery of RGX-314. Anticipated in 2024, the submission of a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) is based on findings from two pivotal trials: ASCENT and the ongoing ATMOSPHERE trial. The ASCENT clinical trial, conducted in collaboration with AbbVie, is expected to enroll participants in both the United States and Canada.

AGTC-501: Beacon Therapeutics

AGTC-501, a gene therapy initiative, is presently undergoing Phase II/III clinical testing to address X-linked retinitis pigmentosa (XLRP). This program became part of Syncona’s portfolio following its acquisition of AGTC in November 2022. XLRP is primarily triggered by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Distinguishing itself from alternative methods, AGTC-501 ensures the expression of the complete RPGR protein, effectively tackling the comprehensive photoreceptor damage associated with XLRP, encompassing both rod and cone loss.

A snapshot of the Gene Therapies In Ophthalmology Pipeline Drugs mentioned in the report:

Gene Therapies In Ophthalmology  Company Phase  Indication
RGX-314 REGENXBIO Phase III Wet age-related macular degeneration
GS010 GenSight Biologics Phase III Leber’s hereditary optic atrophy
SYL1001 Sylentis Phase III Dry eyes
NFS-01 Huazhong University of Science and Technology Phase III Leber’s hereditary optic atrophy
AGTC-501 Beacon Therapeutics Phase II/III Retinitis pigmentosa
ADVM-022 Adverum Biotechnologies Phase II Diabetic retinopathy; Wet age-related macular degeneration
JNJ 81201887 Johnson & Johnson Innovative Medicine Phase II Dry age-related macular degeneration
EXG102-031 Exegenesis Bio Phase I/II Wet age-related macular degeneration
FT-002 Frontera Therapeutics Phase I Retinitis pigmentosa
HG202 HuidaGene Therapeutics Preclinical Age-related macular degeneration

Learn more about the emerging gene therapies in ophthalmology @ Gene Therapies In Ophthalmology Clinical Trials

Scope of the Gene Therapies In Ophthalmology Competitive Landscape Report 

  • Coverage: Global 
  • Key Gene Therapies In Ophthalmology Companies: Spark Therapeutics, Regenxbio, Beacon Therapeutics, Adverum Biotechnologies, Exegenesis Bio, Frontera Therapeutics, HuidaGene Therapeutics, Nanjing IASO Biotherapeutics, GenSight Biologics, Sylentis, Neurophth Therapeutics, Johnson & Johnson Innovative Medicine, Nanoscope Therapeutics, Eyevensys, Atsena Therapeutics Inc., Coave Therapeutics, OCUGEN, INC, Visgenx, Amarna Therapeutics, Ikarovec, Homology Medicines, Ray Therapeutics, Shanghai Refreshgene Technology Co., Ltd., Complement Therapeutics, Abeona Therapeutics, and others
  • Key Pipeline Gene Therapies In Ophthalmology: LUXTURNA, RGX-314, AGTC-501, ADVM-022, EXG102-031, FT 002, HG202, CT103A, GS010, SYL1001, NFS-01, JNJ 81201887, MCO-010, EYS 606, ATSN-201, CTx PDE6B, OCU 400, VGX 0111, AMA 004, IKC159V, Eye disorder Research project, RTx-021, RRG001, CTx 001, ABO-505, and others
  • Company Analysis, Therapeutic Assessment, Pipeline Assessment, Inactive drugs assessment, Unmet Needs

Dive deep into rich insights for new gene therapies in ophthalmology, visit @ Gene Therapies In Ophthalmology 

Table of Contents

1. Gene Therapies In Ophthalmology Pipeline Report Introduction
2. Gene Therapies In Ophthalmology Pipeline Report Executive Summary
3. Gene Therapies In Ophthalmology Pipeline: Overview
4.  Gene Therapies In Ophthalmology Marketed Drugs 
4.1. LUXTURNA: Spark Therapeutics
5. Gene Therapies In Ophthalmology Clinical Trial Therapeutics
6. Gene Therapies In Ophthalmology Pipeline: Late-Stage Products (Pre-registration)
7. Gene Therapies In Ophthalmology Pipeline: Late-Stage Products (Phase III)
7.1. RGX-314: Regenxbio
8. Gene Therapies In Ophthalmology Pipeline: Mid-Stage Products (Phase II/III)
8.1. AGTC-501: Beacon Therapeutics
9. Gene Therapies In Ophthalmology Pipeline: Early-Stage Products (Phase I/II)
9.1. EXG102-031: Exegenesis Bio
10. Gene Therapies In Ophthalmology Pipeline Therapeutics Assessment
11. Inactive Products in the Gene Therapies In Ophthalmology Pipeline
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Unmet Needs
14. Gene Therapies In Ophthalmology Market Drivers and Barriers
15. Appendix

For further information on the gene therapies in ophthalmology pipeline therapeutics, reach out @ Gene Therapies In Ophthalmology Analysis

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