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Bayer and BlueRock Secure FDA Orphan Drug Designation for iPSC Cell Therapy in Retinitis Pigmentosa

23 Jan 2026
Admin
News Article

Bayer and BlueRock Secure FDA Orphan Drug Designation for iPSC Cell Therapy in Retinitis Pigmentosa

Bayer AG and its wholly owned subsidiary BlueRock Therapeutics have received Orphan Drug Designation (ODD) from the US FDA for OpCT-001, an investigational cell therapy for retinitis pigmentosa (RP).

The designation strengthens the clinical and regulatory pathway for a first-of-its-kind iPSC-derived photoreceptor replacement therapy.

Why this matters?

Retinitis pigmentosa is one of the most common inherited retinal disorders. It causes progressive loss of rod and cone photoreceptor cells, leading to irreversible vision loss and, in many cases, legal blindness.

There are no approved therapies that replace lost photoreceptors. OpCT-001 aims to change that.

About OpCT-001

OpCT-001 is an induced pluripotent stem cell (iPSC)-derived cell therapy designed to:

Replace lost photoreceptor cells
Restore retinal function
Potentially improve vision in patients with advanced disease

The therapy is being evaluated in CLARICO, a Phase 1/2a first-in-human clinical study.

Clinical development: CLARICO study design

CLARICO is a multisite, two-part interventional trial enrolling up to ~54 adults with primary photoreceptor diseases, including RP and cone-rod dystrophy.

Phase 1: Safety and dose escalation

Focus: Safety and tolerability
Design: Standard 3+3 dose-escalation scheme
Participants: ~12–24 legally blind adults
Dosing: 4 planned dose levels across 4 cohorts

Dose escalation proceeds only after acceptable safety signals.

Phase 2: Expanded safety and early efficacy

Participants: Up to 30 patients
Design: Randomized 1:1 across two dose cohorts
Endpoints include:
- Visual function
- Functional vision
- Anatomical evidence of cell engraftment

Investigators and patients are masked to dose assignment, except for the surgical team.

Regulatory significance

Orphan Drug Designation is granted to therapies targeting diseases affecting fewer than 200,000 people in the US.

It provides:

Regulatory support and guidance
Potential development incentives
Market exclusivity upon approval

For OpCT-001, it signals FDA recognition of high unmet medical need in inherited retinal disease.

Executive perspective

“This is an important milestone for the OpCT-001 program,” said Dr. Amit Rakhit, Chief Medical Officer at BlueRock. “We believe this therapy has strong potential to restore vision in people living with retinitis pigmentosa.”

Christian Rommel, Global Head of R&D at Bayer Pharmaceuticals, added that the designation reinforces the urgency of advancing innovative therapies for inherited retinal disorders.

Bigger picture: cell therapy moves into ophthalmology

OpCT-001 represents:

The first-ever clinical trial of an iPSC-derived cell therapy for primary photoreceptor diseases
A major expansion of cell therapy beyond neurology and oncology
A long-term bet on regenerative medicine for vision restoration

The program is still investigational. Safety and efficacy have not yet been established. But strategically, this places Bayer and BlueRock at the front edge of regenerative ophthalmology.