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Bayer Begins Phase I/II Study on Cell Therapy for Eye Disease

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Key Takeaways

  • BAYRY has started a phase I/IIa study of OpCT-001 for primary photoreceptor degenerative diseases.
  • OpCT-001 is the first iPSC-derived cell therapy tested in humans for inherited eye conditions.
  • The CLARICO trial assesses safety, vision impact, and cell engraftment across patient subgroups.

Bayer’s (BAYRY - Free Report) wholly owned subsidiary, BlueRock Therapeutics, announced that the first patient in the phase I/IIa CLARICO study has received OpCT-001, an investigational iPSC-derived cell therapy, which is being developed for the treatment of primary photoreceptor diseases.

Per management, OpCT-001 is the first iPSC-derived cell therapy to be tested in humans for treating certain inherited eye diseases like primary photoreceptor diseases. These diseases include conditions like retinitis pigmentosa and cone-rod dystrophy, which can lead to vision loss in children and adults.

OpCT-001 is designed to restore vision in individuals with primary photoreceptor diseases by transplanting functional retinal cells to replace those cells that have degenerated. OpCT-001 offers a promising novel cell-based therapeutic approach aimed at restoring visual function in patients with primary photoreceptor degenerative diseases.

The two-part interventional CLARICO study will evaluate the safety, tolerability and the effect on clinical outcomes of OpCT-001 in patients with primary photoreceptor disease. The phase I portion of the study will focus on safety and includes a dose-escalation design, while the phase II part of the study is designed to collect additional safety data and explore how OpCT-001 impacts visual function, functional vision and anatomical engraftment across different clinical subgroups.

BAYRY's Stock Performance

Year to date, shares of Bayer have rallied 56.6% against the industry’s decline of 0.7%.

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BAYRY's Encouraging Pipeline Progress

Bayer has expanded its pipeline in new modalities to include competencies in cell therapy through the acquisition of BlueRock and in gene therapy through the acquisition of AskBio.

Bayer, together with its subsidiaries, BlueRock and AskBio, is developing preclinical and clinical cell and gene therapies for treating various diseases, including retinal disorders, congestive heart failure and Parkinson's disease, among others.

Last September, the FDA cleared the investigational new drug application for OpCT-001. The regulatory body in the United States granted a Fast Track designation to OpCT-001 for the treatment of primary photoreceptor diseases in February 2025.

The successful development of OpCT-001 could help Bayer redefine therapeutic options for patients facing irreversible vision impairment.

Besides OpCT-001, Bayer, with its wholly owned subsidiary BlueRock, is developing bemdaneprocel (BRT-DA01) in a phase III study for the treatment of Parkinson’s disease. Bemdaneprocel is an investigational stem cell-based therapy that surgically implants dopamine-generating nerve cell precursors into the brain.

The FDA has granted Regenerative Medicine Advanced Therapy designation and Fast Track designation to bemdaneprocel for treating Parkinson’s disease.

BAYRY's Zacks Rank & Other Stocks to Consider

Bayer currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are Exelixis (EXEL - Free Report) , Spero Therapeutics (SPRO - Free Report) and Puma Biotechnology (PBYI - Free Report) , each carrying a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Exelixis’ earnings per share have increased from $2.31 to $2.64 for 2025. During the same time, earnings per share estimates for 2026 have increased from $2.85 to $3.13. Year to date, shares of EXEL have rallied 32.6%.

EXEL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 48.60%.

In the past 60 days, estimates for Spero Therapeutics’ loss per share have narrowed from $2.32 to $1.43 for 2025. During the same time, loss per share estimates for 2026 have narrowed from $1.98 to $1.15. Year to date, shares of SPRO have surged 181.6%.

SPRO’s earnings beat estimates in two of the trailing four quarters, while missing the same on the remaining two occasions, the average surprise being 8.29%.

In the past 60 days, estimates for Puma Biotechnology’s earnings per share have increased from 60 cents to 65 cents for 2025. During the same time, earnings per share estimates for 2026 have risen from 48 cents to 51 cents. Year to date, shares of PBYI have gained 16.1%.

PBYI’s earnings beat estimates in each of the trailing four quarters, the average surprise being 171.43%.

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