Ocugen

Ocugen

OCGNPhase 3
Founded 2013ocugen.com

Ocugen is a clinical-stage biotech focused on developing transformative gene therapies for blindness diseases, with a lead program in Phase 3 for retinitis pigmentosa. The company leverages a unique modifier gene therapy platform designed to address multiple genetic mutations with a single treatment, offering a potential paradigm shift in ophthalmology. Ocugen also maintains a portfolio of inhaled vaccines and biologics, supported by strategic partnerships with Bharat Biotech and CanSinoBIO. The company is led by a seasoned team with deep experience in biopharmaceutical development and commercialization.

Market Cap
$668.9M
Founded
2013
Focus
Biologics

OCGN · Stock Price

USD 2.041.44 (-41.38%)

Historical price data

AI Company Overview

Ocugen is a clinical-stage biotech focused on developing transformative gene therapies for blindness diseases, with a lead program in Phase 3 for retinitis pigmentosa. The company leverages a unique modifier gene therapy platform designed to address multiple genetic mutations with a single treatment, offering a potential paradigm shift in ophthalmology. Ocugen also maintains a portfolio of inhaled vaccines and biologics, supported by strategic partnerships with Bharat Biotech and CanSinoBIO. The company is led by a seasoned team with deep experience in biopharmaceutical development and commercialization.

Technology Platform

A first-in-class modifier gene therapy platform using AAV vectors to deliver nuclear hormone receptors (e.g., NR2E3, RORA) to the retina. This gene-agnostic approach aims to reset retinal homeostasis and treat multiple genetic mutations with a single therapy.

Pipeline Snapshot

10

10 drugs in pipeline, 4 in Phase 3

DrugIndicationStage
Brimonidine Tartrate + PlacebosGraft Versus Host DiseasePhase 3
Brimonidine Tartrate + PlacebosDry EyePhase 3
OCU410STStargardt DiseasePhase 2/3
BBV152COVID-19Phase 2/3
Brimonidine + Brimonidine Mono Therapy + sodium carboxymethylcellulose + Cortico...Dry EyePhase 2

Funding History

3

Total raised: $135M

PIPE$100MUndisclosedFeb 15, 2021
IPO$30MUndisclosedFeb 6, 2014
Series A$5MUndisclosedJun 15, 2013

Opportunities

The primary opportunity is the potential approval and commercialization of OCU400, a first-in-class, gene-agnostic therapy for retinitis pigmentosa, addressing a broad patient population with high unmet need.
Success could validate the modifier gene therapy platform for application in other inherited retinal diseases like Stargardt and dry AMD.
The inhaled vaccine platform offers a differentiated approach in the large and recurring respiratory vaccine market.

Risk Factors

Key risks include clinical failure of the novel modifier gene therapy platform in late-stage trials, regulatory hurdles specific to gene therapies, dependence on raising capital as a pre-revenue company, and intense competition in the ophthalmology therapeutic landscape.
Manufacturing complexities for AAV-based therapies also pose a significant execution risk.

Competitive Landscape

In ophthalmology gene therapy, Ocugen competes with companies like Spark Therapeutics/Roche, Adverum, Regenxbio, and 4DMT. Its key differentiation is the gene-agnostic potential of its modifier gene therapy platform versus mutation-specific approaches. In vaccines, it faces competition from other mucosal vaccine developers. Partnerships with Bharat Biotech and CanSinoBIO provide strategic support.

Publications
19
Patents
7
Pipeline
10

Company Info

TypeTherapeutics
Founded2013
LocationUnited States
StagePhase 3
RevenuePre-revenue

Trading

TickerOCGN
ExchangeNASDAQ

Contact

Therapeutic Areas

OphthalmologyInherited Retinal DiseasesInfectious Diseases

Partners

Bharat BiotechCanSinoBIO (CanSino Biologics Inc.)
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