OpRegen

OpRegen
Clinical data
Trade namesOpRegen
Other namesRG6501
Routes of
administration		Subretinal injection
Legal status
Legal status
  • Investigational

OpRegen (also known as RG6501) is an investigational allogeneic cell therapy developed by Lineage Cell Therapeutics for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).[1][2] The therapy consists of human retinal pigment epithelial (RPE) cells administered via subretinal injection as a single treatment.[3]

Mechanism of action

OpRegen is designed to counteract the dysfunction and loss of retinal pigment epithelial cells that occurs in geographic atrophy by providing cellular support to remaining retinal cells within atrophic areas.[4] The therapy utilizes allogeneic RPE cells derived from human embryonic stem cells through a directed differentiation process, which are then transplanted into the subretinal space to restore function in damaged retinal tissue.[5]

Development and collaboration

OpRegen is currently being developed under an exclusive worldwide collaboration between Lineage Cell Therapeutics, Roche, and Genentech.[1] The therapy represents one of Lineage's three allogeneic "off-the-shelf" product candidates targeting markets with billion-dollar opportunities.[6]

Clinical trials

Phase 1/2a study

The primary Phase 1/2a clinical trial of OpRegen is an open-label, single-arm, multi-center study investigating the safety and tolerability of a single subretinal administration in patients with geographic atrophy.[7] The study includes patients with varying degrees of vision impairment and evaluates both safety parameters and preliminary efficacy measures.[8][9]

The primary objective focuses on evaluating safety and tolerability through assessment of treatment-emergent adverse events, while secondary objectives include evaluating preliminary therapeutic activity by measuring changes in visual function and retinal structure.[8]

Phase 2a study

Following positive Phase 1/2a results, Genentech launched a Phase 2a study of RG6501 (OpRegen) in November 2022, known as the "GAlette study."[1] This advancement represents a significant milestone in the clinical development program for geographic atrophy treatment.[10]

Clinical results

Efficacy outcomes

Clinical trial data have demonstrated promising results for OpRegen therapy. Patients with extensive coverage of OpRegen cell therapy to the geographic atrophy lesion site showed mean vision gains of +9 letters.[4] Evidence of retinal structural improvement has persisted for up to 3 years following treatment, with both anatomical and functional improvements occurring after a single administration.[4]

Safety profile

The therapy has demonstrated a favorable safety profile in clinical trials. OpRegen cells have been shown to survive for extended periods without resulting in teratoma formation or other pathological complications.[11] The cells maintain therapeutic functionality over long periods and have been associated with preserved visual performance in preclinical studies.[11]

Regulatory status

As of 2025, OpRegen continues to advance through clinical development with ongoing data presentations at major scientific conferences. The therapy's 36-month results were featured at Clinical Trials at the Summit 2025, demonstrating continued interest and progress in the field of retinal cell therapy.[12]

References

  1. ^ a b c "Lineage Announces Launch of Phase 2a Study by Genentech of RG6501 (OpRegen®) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration". Business Wire. 28 November 2022.
  2. ^ Yan A, Hasan N, Chhablani J (January 2025). "Dry and neovascular "wet" age-related macular degeneration: Upcoming therapies". Indian Journal of Ophthalmology. 73 (Suppl 1): S55 – S65. doi:10.4103/IJO.IJO_1120_24. PMC 11834902. PMID 39446815.
  3. ^ "OpRegen (RG6501) Phase 1/2a Clinical Study 24-Month Visual Acuity Results Featured at 2024 Retinal Cell & Gene Therapy Innovation Summit". BioSpace. 6 May 2024.
  4. ^ a b c "OpRegen® - Lineage Cell Therapeutics". Lineage Cell Therapeutics. 8 July 2019.
  5. ^ "Anatomical, functional improvements seen with OpRegen in phase 1/2a study". Healio. 20 September 2021.
  6. ^ "Lineage Reports Fourth Case of Retinal Tissue Restoration With OpRegen®". BioSpace. 30 November 2021.
  7. ^ "OpRegen® Phase 1/2a 24-Month Data to Highlight 2024 Retinal Therapy Summit". Synapse Patsnap.
  8. ^ a b "RG6501 (OpRegen®) Phase 1/2a Clinical Study 24 Month Results to Be Featured at 2024 Retinal Cell & Gene Therapy Innovation Summit". Business Wire. 13 March 2024.
  9. ^ Hoffmann-La Roche (4 July 2025). Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients With Advanced Dry-Form Age-Related Macular Degeneration (Geographic Atrophy) (Report). clinicaltrials.gov.
  10. ^ Genentech, Inc. (4 July 2025). A Phase IIa, Multicenter, Open-Label, Single-Arm Study To Optimize Subretinal Surgical Delivery And To Evaluate Safety And Activity Of Opregen In Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (Report). clinicaltrials.gov.
  11. ^ a b "RPE Cell-Based OpRegen Receives Positive Results in Preclinical Studies". The American Society of Retina Specialists.
  12. ^ "RG6501 (OpRegen®) Phase 1/2a Clinical Study 36 Month Results to Be Featured at Clinical Trials at the Summit 2025". Business Wire. 12 May 2025.
This article is issued from Wikipedia. The text is available under Creative Commons Attribution-Share Alike 4.0 unless otherwise noted. Additional terms may apply for the media files.