Cell therapy weekly: Rutgers researchers introduce an innovative approach to treating spinal cord injury
This week: Rutgers researchers introduce an innovative approach to treating spinal cord injury, Novartis and Voyager Therapeutics enter agreement for AAV gene therapy vectors and US Congressman introduces bill to improve safety of accelerated drug approvals.
The news highlights:
AI and robotics: Rutgers researchers’ innovative approach to treating spinal cord injury
A team led by Rutgers University (NJ, USA) researchers has successfully stabilized the enzyme ChABC, which is able to degrade spinal cord injury (SCI) scar tissue. ChABC promotes degradation of scar tissue molecules and the regeneration of tissue, however, it is highly unstable at the human body temperature of 96.8°F. The study employed an artificial intelligence-driven approach utilizing liquid handling products to synthesize and test the copolymers that wrap around and stabilize ChABC in hostile microenvironments. One copolymer combination provided promising results for patients recovering from SCI, as it was able to retain 30% of the enzyme for up to 1 week.
“This study represents one of the first times artificial intelligence and robotics have been used to formulate highly sensitive therapeutic proteins and extend their activity by such a large amount. It’s a major scientific achievement,” remarked Adam Gormley, principal investigator and an assistant professor of biomedical engineering at Rutgers School of Engineering at Rutgers University.
Novartis and Voyager Therapeutics enter agreement for AAV gene therapy vectors
Novartis (Basel, Switzerland) and Voyager Therapeutics (MA, USA) have entered a strategic agreement utilizing Voyager’s TRACER adeno-associated virus (AAV) capsids in potential gene therapies for three neurological diseases. The agreement will see Novartis invest US$54 million, with another US$1.7 billion potentially available to Voyager for exercise fees and milestone payments. With access to Voyager’s capsids, Novartis aims to expand its impact in the treatments of neurological disease, where commonly used vectors have low efficacy.
“The challenge of biodistribution of AAV gene therapies to target cell types in the brain is longstanding, but we refuse to believe it is insurmountable. We see the potential of Voyager’s innovative platform to imagine new types of therapeutics for life-threatening CNS diseases,” stated Jay Bradner, president of Novartis Institutes for BioMedical Research.
US Congressman introduces bill to improve safety of accelerated drug approvals
A US bill has been introduced by Congressman Frank Pallone Jr. to grant the FDA greater power in ensuring that drug approvals have proven clinical benefit. The proposed Accelerated Approval Integrity Act aims to streamline the process of taking products off the market if they provide no clinical benefit, a response that supports the FDA’s existing accelerated approval pathway. The pathway has procedures that allows drugs without sufficient clinical value to be pulled from market, however, these procedures are resource-intensive and seldom used.
The bill would enforce a more structured method of approval, making it mandatory for manufacturers to enter an agreement with the FDA on how the study will be undertaken and ensuring that confirmatory studies are in the pipeline. Accelerated approval may also be withdrawn in the circumstance that the study fails to meet the targets of the study that were agreed upon enrollment.
“Patients deserve to know that the drugs they are taking are safe and effective…[but] under the current system, some products have been allowed to stay on the market for far too long without clinical trials that demonstrate a real clinical benefit,” commented Frank Pallone Jr., US Congressman and chair of the House Energy and Commerce Committee.
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