Drug intelligence / Profile preview

bemdaneprocel

Development stage
Phase 3
Lead developer
BlueRock Therapeutics
Modality
Stem Cell Therapies → Cell Therapies
Administration
Surgical Implantation, Intracerebral
01

Overview

Bemdaneprocel (BRT-DA01) is an investigational, off-the-shelf, allogeneic cell therapy designed to treat Parkinson's disease by replacing lost dopamine-producing neurons. The therapy consists of midbrain dopaminergic neuron progenitor cells derived from human embryonic stem cells (hESCs). These cells are surgically implanted into the post-commissural putamen, where they mature into functional neurons that integrate into the brain's circuitry and provide a sustained source of dopamine. Developed by BlueRock Therapeutics (a subsidiary of Bayer) in collaboration with Memorial Sloan Kettering Cancer Center, bemdaneprocel has received FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations. In Phase 1 trials, it demonstrated safety, tolerability, and evidence of graft survival and motor symptom improvement, leading to the initiation of a registrational Phase 3 trial (exPDite-2).

Other names
human embryonic stem cell-derived midbrain dopamine neuron cell therapy

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