Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation


NeoCart was originally developed by the company Histogenics.

Ocugen has added the phase 3 cell therapy platform NeoCart to its pipeline following its Regenerative Medicine Advanced Therapy (RMAT) recognition for the repair of full-thickness lesions of the knee cartilage in adults.1

“We’re excited that NeoCart® has received this RMAT designation, an important regulatory milestone, especially as we view this product as an enabling technology in cell and regenerative therapy for orthopedic indications. Our next step will be working with the FDA to construct the Phase 3 program to bring this innovation to this emerging treatment area,” Shankar Musunuri, PhD, Chairman, chief executive officer and co-founder, Ocugen, said in a statement.1

Neocart consists of autologous chondrocyte-derived neocartilage and was acquired as part of Ocugen’sreverse merger with Histogenics in 2019. The cell therapy is intended to repair articular cartilage lesions which may limit mobility and lead to osteoarthritis.

“People living with articular cartilage lesions literally have holes in their knees that are extremely difficult to heal, and without proper treatment, they’re at high risk of getting osteoarthritis. We believe that NeoCart® offers the potential for an innovative new option where treatments in this area are still limited and results are not optimal,” Musunuri added to the statement.1

WATCH NOW: Harnessing Gene Therapy for Osteoarthritis

Ocugen also recently announced in April 2022 that they had received a positive Data Safety Monitoring Board (DSMB) recommendation for their phase 1/2 clinical trial (NCT05203939) assessing OCU400, a gene therapy for the potential treatment of retinitis pigmentosa.2 The DSMB has recommended that Ocugen continue enrolling participants in the current cohort at the target dose level.

OCU400 targets nuclear hormone receptors that regulate multiple functions in the retina, which may be able to address multiple gene mutations and multiple retinal diseases with 1 therapy. The phase 1/2 study is primarily assessing safety as measured by the incidence and severity of adverse events (AEs). Secondary endpoints include best-corrected visual acuity, low-luminance visual acuity, slit-lamp biomicroscopy, intraocular pressure, indirect ophthalmoscopy, anti-adeno associated virus vector antibodies, anti-hNR2E3 antibodies, and T-cell response. Additional outcomes will assess other measures of vision improvements.

“It’s a positive first step that the DSMB review of the current OCU400-101 study results identified no serious adverse events and recommended that the study proceed with enrollment,”OcugenRetina Scientific Advisory Board member Mark Pennesi, MD, PhD, professor of ophthalmology and chief, Paul H. Casey Ophthalmic Genetics Division, Oregon Health & Science University, said in a previous statement.2 “We’re looking forward to understanding how this modifier gene therapy platform could treat inherited retinal degeneration, potentially bringing an option to people affected with this disease.”

1. Ocugen announces new cell therapy program following FDA Regenerative Medicine Advanced Therapy (RMAT) recognition. News release. Ocugen. May 24, 2022.
2. Ocugen, Inc. announces positive DSMB recommendation for OCU400-101 clinical trial. News release. April 25, 2022.
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