Ocugen Advances Toward Key Regulatory Submission for Gene Therapy

Ocugen outlines FDA pathway for OCU400, a one-time gene therapy for Retinitis Pigmentosa, with BLA targeted for H1 2026 and $20.85M raised to fund development.

Biopharmaceutical firm Ocugen, Inc. has outlined a definitive regulatory pathway for its lead gene therapy candidate. The Malvern-based company is preparing to initiate a rolling Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for OCU400 within the current year. This process is targeted for completion by the first half of 2026, marking a pivotal phase for the potential treatment of Retinitis Pigmentosa.

Broad Therapeutic Strategy Shows Promise

The OCU400 program is notable for its ambitious, mutation-agnostic approach. The therapy is designed as a one-time injection intended to address the numerous genetic mutations responsible for causing Retinitis Pigmentosa. This strategy could prove transformative for a significant patient population that currently lacks approved treatment options.

Early clinical data has provided a foundation for optimism. In a study involving 18 participants, all treated subjects demonstrated either an improvement or a stabilization in visual function over a two-year observation period when compared to their untreated eyes. Investigators reported the therapy’s safety and tolerability profile as favorable.

Capital Position Strengthened for Critical Period

Ocugen has fortified its balance sheet to support this crucial development stage. The company concluded a public offering in late January 2026, raising net proceeds of approximately $20.85 million. Management anticipates these funds will be sufficient to finance operations through the fourth quarter of 2026, aligning with the anticipated timeline for the BLA submission and review.

Market participants will gain further insight into the company's strategy with the upcoming earnings release. Ocugen is scheduled to report its fourth quarter and full-year 2025 financial results on March 4, 2026, where additional operational details are expected. The company has already seen increased institutional investment in its shares during the last quarter, with a notable announcement on February 25, 2026, providing further positive momentum for its stock.