Ikthera is a Bengaluru-based biotechnology innovation company established to advance scientifically differentiated therapies from validated discovery programmes into regulated clinical development in India. Its initial programme, AAV.CRYBA1, is being prepared for first-in-human evaluation in geographic atrophy secondary to dry age-related macular degeneration.
How we work
GROW Research Laboratory at Narayana Nethralaya Foundation serves as the on-ground scientific and laboratory partner for Ikthera. All programme research, vector development, analytical work and preclinical execution are performed through GROW/NNF; Ikthera does not maintain a separate laboratory footprint. Ikthera will remain the long-term clinical-translation vehicle, since GROW and NN cannot be commercial entities, thus coordinating regulatory development, national clinical partnerships, multicentre expansion and future access across India, subject to the applicable approvals and quality requirements.
On-ground scientific and laboratory execution
Ophthalmic clinical interface and long-term follow-up
CMC, nonclinical and clinical development coordination
Multicentre partnerships, access and future adoption
Unmet need and development rationale
India has both a meaningful present burden of age-related macular degeneration and a rapidly ageing population. A population-based study in rural and urban north and south India found late AMD in 1.2% of adults aged 60–79 years and 2.5% of those aged 80 years or older; earlier AMD changes were substantially more common. The investigators cautioned that estimates in the oldest group may be conservative because non-attendance and cataract-related ungradability were greater.
1.2%
Late AMD in adults aged 60–79 in India
2.5%
Late AMD in adults aged 80 and older
India population aged 60+
% of population
People living with AMD worldwide
millions
The number of older adults in India is projected to increase markedly, making retinal-degeneration preparedness a public-health and health-system priority.
GROW/NNF combines AAV engineering, ocular models, analytical development, retinal imaging and specialist clinical care within one India-based translational ecosystem.
A safety-led ocular gene-therapy programme requires durable clinical engagement and harmonised imaging, functional and laboratory assessments that is available at Narayana Nethralaya.
Local development strengthens domestic control over vector know-how, assays, trained personnel and eventual supply-chain decisions, subject to GMP and regulatory qualification.
Beyond India
AMD affected an estimated 196 million people in 2020 and is projected to affect about 288 million by 2040. Geographic atrophy causes irreversible loss of RPE and photoreceptors, and current complement-directed therapies slow lesion enlargement without restoring lost tissue and require repeated intravitreal treatment. A durable, RPE-directed therapy developed in India could therefore address a global need if human safety, clinically meaningful activity, durability, scalable manufacture and acceptable cost are demonstrated.
Worldwide adoption is a development objective, not an established outcome.
A differentiated scientific foundation
The programme is built on a mechanistic body of work identifying βA3/A1-crystallin, encoded by CRYBA1, as a regulator of RPE lysosomal function, cellular clearance and inflammatory homeostasis rather than as a conventional lens-only structural protein.
βA3/A1-crystallin localises to lysosomes and interacts with the V-ATPase machinery. Conditional loss in RPE raises lysosomal pH, reduces proteolytic activity and impairs autophagic flux.
CRYBA1 deficiency perturbs SLC36A4/PAT4-linked amino-acid sensing, mTORC1 signalling and TFEB/CLEAR network activity, providing a mechanistic bridge between lysosomal stress and deposit-forming RPE phenotypes.
CRYBA1-deficient models show LCN2-associated innate immune signalling, neutrophil recruitment and AKT2-linked inflammatory dysregulation; complementary studies connect LCN2 with impaired autophagy and inflammasome-ferroptotic injury.
βA3/A1-crystallin regulates apical polarity and EGFR endocytic trafficking, processes relevant to maintenance of a functional RPE monolayer.
Therapeutic hypothesis
Restoring CRYBA1 expression selectively in RPE may improve lysosomal acidification and clearance, reduce maladaptive inflammatory signalling and preserve RPE structure and function. AAV delivery provides sustained local expression from a single administration, subject to confirmation of dose, biodistribution, durability and safety.
This scientific foundation reflects a multi-year collaboration between Prof Debasish Sinha’s group and Dr Arkasubhra Ghosh’s GROW team, connecting mechanistic discovery with disease models, vector design, ocular delivery and regulated translation in India.
India-based research and laboratory execution
All research and laboratory activities for the Ikthera programme are performed through GROW Research Laboratory and Narayana Nethralaya Foundation in Bengaluru. Ikthera has no independent laboratory footprint. This role separation places experimental execution, analytical work and facility governance within the specialist institution that generates the data.
Capsid, promoter, transgene and plasmids
AAV capsid/transgene design, promoter selection, plasmid systems, small-scale screening and RPE-targeted expression assessment.
Upstream, downstream, formulation and scale-up
Upstream production including fixed-bed approaches, downstream purification, formulation development, scale-up strategy and process analytics.
Identity, titre, purity, potency and stability
Identity, genome titre, purity, residuals, infectivity or expression-linked potency, sterility-related controls and stability-method development, as applicable.
Models, imaging, immunology and biodistribution
Rodent and large-animal studies, suprachoroidal delivery, multimodal retinal imaging, electrophysiology, histology, immunology and biodistribution.
Retina specialists, sites, follow-up and data systems
Direct interface with a high-volume tertiary ophthalmology network, trained retinal specialists, imaging infrastructure and clinical-research operations.
Integrated on-ground capabilities supporting the Ikthera AAV.CRYBA1 programme at GROW/NNF.
Relevant expertise
The programme draws on complementary expertise in India-based clinical translation, dry-AMD biology, ocular AAV development and biotechnology strategy.
Director, Ikthera
Provides India-based programme stewardship and coordinates institutional, regulatory and clinical-development interfaces. His operational leadership at Narayana Nethralaya supports a practical pathway from laboratory development to national clinical translation.
Programme strategy and technology translation
Entrepreneur and computer scientist working across genetics, artificial intelligence and biotechnology; supports programme strategy, innovation and translation.
Gene therapy research advisor
Founder of GROW; leads ocular AAV design, translational studies and India-based vector-development capability, with experience contributing to national gene-therapy guidance.
CRYBA1 biology and dry AMD science research advisor
Johns Hopkins ophthalmology scientist whose work established the lysosomal, autophagy and inflammatory relevance of CRYBA1 in RPE and dry-AMD models.
Ikthera retains long-term responsibility for programme coordination and national clinical translation. GROW/NNF remains the on-ground laboratory and scientific execution partner, while the scientific leads remain responsible for evidence generation and interpretation within the relevant governance and quality systems.
Get in touch
Ikthera Therapeutics India Private Limited
No 31 SLN Nilaya, 2nd Main, Gopal Reddy Layout,Banaswadi, Bangalore North, Bangalore – 560043
CIN: U21009KA2025FTC203254