Gene Therapy in Ophthalmology Market SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2024 - 2031)

Gene Therapy in Ophthalmology Market Size - Analysis

Gene Therapy in Ophthalmology Market Trends

Genetic factors contribute significantly to the development of various ophthalmic conditions including retinitis pigmentosa, age-related macular degeneration (AMD), and glaucoma. Researchers have found that mutations in as many as 250 genes can result in retinal degeneration alone. The worldwide prevalence of such inherited retinal diseases (IRDs) is estimated to range from 1 in 2,000–3,000 persons.

Gene therapies, however, offer the promise of long-term vision restoration or preservation by precisely targeting the underlying genetic roots of ocular disorders. This potential for gene therapies to provide a curative outcome through a single administration has spurred significant interest from biopharmaceutical firms. It has also encouraged greater patient enrollment in ongoing clinical trials evaluating candidate therapies for conditions like Leber congenital amaurosis and AMD.

Market Driver - Advances in Gene Editing Technologies Enhancing the Development of Novel Therapies

Building on these platforms, scientists are gaining deeper insights into disease-causing gene mutations and the complex molecular mechanisms underlying retinal degeneration. This has led to the creation of several proof-of-concept animal models that emulate human genetic eye diseases. Such models have been instrumental in demonstrating proof-of-principle for novel therapeutic strategies like knocking-out defective genes, inserting functional gene copies and gene supplementation through viral vector delivery.

Market Challenge - High Costs Associated with Gene Therapy Treatments Limiting Accessibility

Additionally, manufacturing gene therapies is a complex process that often involves customized viral vectors and careful dosing for each patient's specific needs. All of these factors contribute to the high price tags of many gene therapies currently available or in the pipeline.

As gene therapies for ophthalmic conditions continue to advance, bringing down the costs associated with development as well as administration will be crucial to maximize the utilization and benefits of this technology across broader patient populations.

Market Opportunity - Expansion into Emerging Markets with Unmet Medical Needs

Gene therapies have the potential to transform the management of genetically-driven retinal disorders that currently have limited treatment options in these markets. International drug developers and domestic pharma companies can capitalize on this unmet need by conducting clinical trials and regulatory submissions strategically tailored for emerging markets. Partnering with local healthcare providers and patient advocacy groups will help facilitate patient identification and treatment delivery.

Key winning strategies adopted by key players of Gene Therapy in Ophthalmology Market

Novartis has been a pioneer in developing gene therapy for ophthalmology indications. In 2017, it received approval for Luxturna (voretigene neparvovec), the first ever gene therapy approved in the US for an inherited retinal disease. Luxturna is designed for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It helped restore vision in patients who would have otherwise experienced progressive vision loss. Luxturna’s approval demonstrated the promise of gene therapy for treating inherited eye conditions. It gave Novartis first-mover advantage and established its leadership in this therapeutic area.

Both companies’ successes reinforced gene therapy as a viable treatment paradigm for ophthalmic conditions like inherited retina diseases. They established clinical proof-of-concept which de-risked the field and attracted more players. Over 15 biotechs are now developing various gene therapies for retinal degenerations, glaucoma, dry eye etc.

Segmental Analysis of Gene Therapy in Ophthalmology Market

In terms of vector type, adeno-associated virus (AAV) segment is estimated to hold 64.5% share of the market in 2024, due to its excellent clinical potential for ocular gene therapy. AAV vectors have emerged as the leading vector of choice owing to their non-pathogenic nature and ability to efficiently transduce retinal cells without adverse effects. They also have the advantage of long-term transgene expression in post-mitotic cells like photoreceptors.

The scalability of AAV vector production and longer stability also offer economic and logistical benefits. Their proven ability to safely deliver therapeutic genes makes AAV the most widely used and commercially viable vector system currently for retinal gene therapy.

In terms of end user, hospitals contribute the highest share of the market owing to the need for advanced infrastructure and multidisciplinary care required for gene therapy procedures. Gene therapy for ocular disorders is a complex treatment modality requiring specialized equipment, trained professionals, and biosafety facilities for vector production and administration.

The in-patient hospital setting also allows for postoperative care and monitoring of patients. As gene therapy demand increases with commercialization, hospitals will play a central role in its delivery utilizing their advanced facilities and wider network to improve patient access to these novel sight-saving therapies. Ambulatory surgery centers and specialized eye clinics may also contribute to the hospital segment growth over the long term.

Additional Insights of Gene Therapy in Ophthalmology Market

• Over 200,000 individuals worldwide are affected by inherited retinal diseases, representing a significant market opportunity.
• Gene therapies have the potential to provide long-term solutions with a single administration, reducing the treatment burden.
• The market saw its highest share in the United States in 2020, with approximately USD 35 million, followed by Germany within the EU4.
• In Japan, there were around 800,000 cases of wet AMD eligible for gene therapy in 2020.
• The successful approval and commercialization of Luxturna have set a precedent, encouraging other companies to invest in gene therapies for ophthalmic diseases.

Competitive overview of Gene Therapy in Ophthalmology Market

The major players operating in the gene therapy in ophthalmology market include Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neuropht Therapeutics, Adverum Biotechnologies, EyeVensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, REGENXBIO, Skyline Therapeutics, Huida Gene Therapeutics, Opus Genetics, Spark Therapeutics, Novartis AG, Adverum Biotechnologies, REGENXBIO Inc., and AGTC.

Recent Developments in Gene Therapy in Ophthalmology Market

Gene Therapy in Ophthalmology Market Segmentation

• By Indication
  • Inherited Retinal Diseases
  • Leber Congenital Amaurosis
  • Retinitis Pigmentosa
  • Choroideremia
  • Acquired Retinal Diseases
  • Age-related Macular Degeneration
  • Diabetic Retinopathy
• By Vector Type
  • Adeno-associated Virus (AAV)
  • Lentivirus
  • Others
  • Retrovirus
  • Herpes Simplex Virus
• By End User
  • Hospitals
  • Ophthalmic Clinics
  • Academic and Research Institutes
• By Route of Administration
  • Subretinal Injection
  • Intravitreal Injection
  • Others