Phase III trial for Parkinson’s disease cell therapy
The first patient has been treated in a pivotal phase III clinical trial of bemdaneprocel (BRT-DA01), an investigational cell therapy for Parkinson’s disease. The therapy is being developed by BlueRock Therapeutics, a subsidiary of Bayer AG, and has received FDA Fast Track and Regenerative Medicine Advanced Therapy designations.
Bemdaneprocel is designed to replace the dopamine-producing neurons lost in Parkinson’s disease. Derived from human embryonic pluripotent stem cells, the therapy involves implanting dopaminergic neuron precursors into the brain, where they are expected to mature and integrate into neural networks disrupted by the disease.
The main goal of the trial is to measure whether patients experience more time during the day when their medication is working effectively (known as ON-time) without causing troublesome involuntary movements. This will be tracked using patient diaries over a period of 78 weeks. In addition, the study will look at a range of other outcomes, including changes in movement and non-movement symptoms of Parkinson’s disease, the safety and tolerability of the therapy, and how the treatment affects daily functioning and overall quality of life as reported by patients.
New European guidelines for variations
The European Commission has published new Variations Guidelines that will take effect in January 2026. The guidelines, developed with support from the EMA and the European medicines regulatory network, are intended to simplify and harmonize the management of post-approval changes to medicines. The new framework introduces a clearer risk-based classification system for variations, distinguishing between minor changes with limited impact, minor changes requiring notification, and major changes with potential implications for quality, safety, or efficacy. It also formalizes the use of regulatory tools such as Post-Approval Change Management Protocols and Product Lifecycle Management Documents to support more predictable and efficient lifecycle management.
Marketing authorization holders will be required to follow the new guidelines from 15 January 2026. Ahead of that deadline, EMA will provide updated procedural guidance by the end of 2025 to help companies prepare for implementation.
Pfizer to acquire Metsera
Pfizer has entered into a definitive agreement to acquire Metsera, a clinical-stage biopharmaceutical company focused on obesity and cardiometabolic diseases. The deal is valued at $47.50 per Metsera share in cash at closing, representing an enterprise value of approximately $4.9 billion, with the potential for an additional $22.50 per share tied to clinical and regulatory milestones. The transaction has been unanimously approved by both companies’ boards and is expected to close in the fourth quarter of 2025, subject to customary approvals.
Through the acquisition, Pfizer will add four clinical-stage programs to its pipeline, including oral and injectable incretin and non-incretin therapies. The portfolio features MET-097i, a GLP-1 receptor agonist in phase II developmen, and MET-233i, an amylin analog in phase I studies. In addition, Metsera is advancing oral GLP-1 receptor agonist candidates expected to enter the clinic shortly.
Funding more effective pharmaceutical catalysts
The University of Virginia and Johns Hopkins University have received funding from the US National Science Foundation to develop more effective catalysts for pharmaceutical manufacturing. The collaboration, led by Jason Bates at Virgina and Brandon Bukowski at Johns Hopkins, is focused on stabilizing molecular catalysts by anchoring them to solid supports such as zeolites. This approach is expected to extend catalyst lifetimes, limit side reactions, and improve recovery and reuse, thereby enhancing product quality and process efficiency.
A key objective of the project is to make continuous-flow manufacturing more practical in pharmaceutical settings. By improving catalyst stability and performance, the research aims to reduce downtime, minimize waste, and lower costs associated with catalyst replacement and by-product management. The work integrates computational modeling with experimental validation to better understand catalyst behavior at the molecular level.
Reducing the error rate of prime editing
MIT researchers have developed a new method to reduce the error rate of prime editing. The team introduced specific Cas9 mutations that loosen strictness in cut location and developed an enhanced RNA template stabilization. Their published work shows that the error rate in the most commonly used editing mode was reduced from one in seven edits to around one in 101. In a higher precision editing mode, errors dropped from one in 122 edits to about one in 543.
The team is now working on further improving the efficiency of prime editors through further modifications of Cas9 and the RNA template. They are also working on ways to deliver the editors to specific tissues of the body, which is a longstanding challenge in gene therapy.
Broermann Medical Innovation Award for Carl June and Sadelain
Carl June and Michel Sadelain have been awarded the 2025 Broermann Medical Innovation Award by Asklepios Kliniken GmbH & Co KGaA in recognition of their groundbreaking contributions to the development of CAR T cell therapy. June and Sadelain’s work established the scientific and translational framework for CAR T cell therapy. Their contributions include defining key elements of receptor design, demonstrating clinical feasibility, and leading pivotal studies that have informed both regulatory pathways and broader adoption in practice.
The award will be formally presented in Germany later this year.
$53 million in funding for Ensoma
Ensoma has completed a $53 million financing round to advance its lead program EN-374 and continue development of its in vivo hematopoietic stem cell engineering platform. The funding will support progress through key clinical milestones, including the ongoing phase I/II study of EN-374 in X-linked chronic granulomatous disease (X-CGD).
EN-374 is designed as a first-in-class in vivo therapy that uses virus-like particles to deliver a functional CYBB transgene into hematopoietic stem cells, enabling the generation of neutrophils with restored NADPH oxidase activity. Preclinical data demonstrated restoration of CYBB expression and oxidase function in animal models, providing the basis for clinical evaluation. Beyond EN-374, the financing will also support expansion of Ensoma’s platform into additional indications, including immuno-oncology and sickle cell disease.
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