This week: BlueRock Therapeutics (MA, USA), a wholly owned and independently operated subsidiary of Bayer AG (Leverkusen, Germany), treated its first patient in a Phase III Parkinson’s disease trial. Meanwhile, the push to advance cell and gene therapy continues, with companies forming strategic partnerships to expand development in Southeast Asia and the launch of a promoter library aimed at enhancing natural killer (NK) cell immunotherapies.
The news highlights:
First patient receives stem cell therapy in Phase III Parkinson’s disease trial
The first patient has been treated in BlueRock Therapeutics’ Phase III trial, exPDdite-2, which is evaluating bemdaneprocel (BRT-DA01), an investigational allogeneic pluripotent stem cell-derived therapy for Parkinson’s disease. BRT-DA01 has been designed to replace the dopamine-producing neurons lost in Parkinson’s disease. These dopaminergic neuron precursors, derived from human embryonic pluripotent stem cells, are implanted into the brain during surgery. After transplantation, they develop into mature dopamine neurons, with the potential to rebuild damaged neural networks and restore both motor and non-motor functions in patients.
“People living with Parkinson’s disease urgently need new therapies that truly alter the course of the disease,” said Amit Rakhit, BlueRock’s Chief Development and Medical Officer. “The initiation of the exPDite-2 trial represents a major step forward toward advancing bemdaneprocel and we are excited to build on the momentum of our earlier data to further develop it as a potentially transformative cell therapy aimed at restoring motor and non-motor function.”
BlueRock plans to enroll approximately 102 patients with Parkinson’s disease in exPDite-2, a multicenter, double-blind trial designed to evaluate the efficacy, safety, and overall impact of BRT-DA01 compared to a placebo surgery control.
Partnership expanding cell and gene therapy development in Southeast Asia
Contract Development and Manufacturing Organization eXmoor Pharma (Bristol, UK) and biopharmaceutical company Siam Bioscience (Bangkok, Thailand) have partnered to establish a cell and gene therapy development center at Siam Bioscience’s manufacturing site in Bangkok, Thailand. This collaboration aims to accelerate the growth of cell and gene therapy in Thailand and Southeast Asia by helping therapy developers deliver advanced medicines efficiently and in compliance with regulatory standards. The partnership will offer comprehensive services, including consultancy, process and analytical development, as well as GMP clinical and commercial manufacturing.
“We are pleased to collaborate with Siam Bioscience on such a visionary and impactful initiative. Thailand and Southeast Asia are rapidly emerging as hubs for cell and gene therapy innovation,” commented Angela Osborne, Executive Chair of eXmoor Pharma. “By combining Siam Bioscience’s regional leadership with eXmoor’s technical expertise and global [Contract Development and Manufacturing Organization] capabilities, we are creating a launchpad to support both local breakthroughs and international partnerships. This is about bringing life-changing therapies to more patients, more quickly, and with the right infrastructure to support long-term success.”
Launch of a promoter library to enhance NK cell immunotherapies
Biotechnology company SynGenSys (Sheffield, UK) has introduced NK.SET™, a synthetic promoter library designed to enhance the precision and tunability of gene expression in NK cell immunotherapies, improving both efficacy and targeting. Each promoter is engineered to minimize off-target activity and can be customized to meet specific therapeutic needs. The library offers ready-to-use targeted promoters and serves as a foundation for rapid customization, optimized for individual therapeutic applications.
“The introduction of NK.SET marks a significant step forward for SynGenSys, and a clear demonstration of the utility of our platform,” said David James, Co-Founder and Chief Scientific Officer of SynGenSys. “Not only does it open the door for new partnership opportunities in the expanding NK-based immunotherapy field, but it also extends the potential for applicability of our platform toother immune cells such as T cells, broadening our impact across the cell and gene therapy landscape. This is more than a product – it’s a strategic milestone showcasing SynGenSys’ commitment to advancing the next-generation of cell and gene therapies through engineered transcriptional control – ultimately delivering safer and more effective therapies.”
This was shown first on: https://www.regmednet.com/cell-therapy-weekly-first-patient-receives-stem-cell-therapy-in-phase-iii-parkinsons-disease-trial/
