This Week’s CGT News: Vinay Prasad Becomes New Director of FDA CBER
Our latest news roundup reports on Vinay Prasad’s appointment, a personalized gene therapy for a baby with an ultra rare condition, and more
| 3 min read | News
Vinay Prasad to succeed Peter Marks
Vinay Prasad has been appointed as the new director of FDA’s CBER, where he will oversee the regulation of vaccines, gene therapies and blood products. He replaces Peter Marks, who resigned from the agency in March.
Prasad is a hematologist-oncologist and a professor at the University of California, San Francisco. He has authored over 500 academic articles and two books that critique aspects of medical evidence and regulatory practices. He has persistently advocated for high quality evidence and greater transparency in regulatory standards.
However, he has also sparked controversy by being a highly outspoken critic of many aspects of public health and medical regulation. For example, he has spoken out against COVID-19 vaccines for children and mask mandates. In October 2021, Prasad published a blog post warning that US pandemic policies, such as emergency powers and suppression of dissent, could pave the way for authoritarianism.
Speaking on the social media platform X, FDA Commissioner Martin Makary said, “Dr. Prasad brings the kind of scientific rigor, independence, and transparency we need at CBER—a significant step forward.”
Personalized gene therapy for ultra-rare condition
Andelyn Biosciences has used its AAV Curator platform to manufacture a novel gene therapy for a baby diagnosed with NEDAMSS (neurodevelopmental disorder with regression, abnormal movements, loss of speech, and seizures), an ultra-rare monogenic condition that has no known cures or treatments. The therapy was produced and released 10 months from the start of development and 14 months after the patient's initial diagnosis.
One month post-treatment, the therapy has been safe and well tolerated but it is too early to assess efficacy.
Andelyn’s platform facilitated the development of the AAV-based gene therapy by streamlining vector selection, design, and production, in collaboration with the Columbus Foundation and Nationwide Children’s Hospital. The patient-specific therapy is part of an IND-authorized program and reflects a targeted approach to address diseases with extremely limited treatment options.
In a statement, Wade Macedone, CEO of Andelyn Biosciences, said: “Supporting single-patient projects is part of the DNA of Andelyn Biosciences and the lifestyle of everyone who works at the organization. As a company we are as proud of successfully completing single-patient programs as we are scaling a manufacturing process for 10,000 or more patients. Individually, we all know that with these projects there is someone, usually a young child, whose life depends on us.”
New technology launched by Univercells
Univercells Technologies, a Donaldson Life Sciences company, has announced the release of the scale-X nitro controller, a new bioprocessing system designed to operate its 200 m² and 600 m² scale-X nitro bioreactors. The controller is developed for use in the production of viral vectors, vaccines, and other advanced therapies, supporting both adherent and suspension cell cultures.
It is engineered to facilitate integration into existing manufacturing environments, offering a compact footprint of 1.6 m² and a height of under two meters. According to the company, the controller is intended to support rapid scale-up from research through to commercial manufacturing, with the potential to reduce production timelines and associated infrastructure demands. The device also aims to address cost efficiency, with cited reductions in manufacturing costs compared to conventional adherent cell culture platforms.
The introduction of the nitro controller is positioned by Univercells Technologies as the final component in its scale-X platform, completing a modular offering for intensified viral and therapeutic protein production.
Partnership to push CAR T for glioblastoma
CDMO Cellipont Bioservices and Optieum Biotechnologies, a preclinical stage company working on CAR T therapies, have agreed a partnership for the cGMP manufacturing of Optieum’s investigational CAR T cell therapy for glioblastoma. OPTF01 is based on Optieum's Eumbody System, which is designed to enhance CAR-T function by harmonizing the activity of single-chain variable fragment (scFv) domains. The therapy targets Fibroblast Activation Protein-alpha (FAPα), a protein expressed both on glioblastoma tumor cells and within the tumor stroma, enabling a dual mechanism of action intended to address tumor cells and the surrounding immunosuppressive environment.
Under the terms of the collaboration, Cellipont Bioservices will manage the technology transfer, process development, and cGMP manufacturing of the OPTF01 cell therapy.
