Regenerative Medicine

Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.

Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.

With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.

US and EU regulators have invited two companies to work with them on a pilot scheme for assessing drugs for rare genetic diseases, a move that the European Medicines Agency’s Emer Cooke said would help improve “global alignment.”

Exosome-based treatments hold promise, but their reality has not yet materialized in the form of compelling clinical data. Belgian biotech Exo Biologics’ CEO Hugues Wallemacq believes his company can help move the field forward, both through its own clinical programs and by offering high-quality exosomes to others in the field.   

In a video interview with Scrip, Syed Husain, CEO of the US-based cell therapy CDMO BioCentriq, talks about the company’s role in parent GC’s growth strategy and business priorities, the cell and gene therapy manufacturing market and his views on the US BIOSECURE Act.

An FDA question to a patient concerned about the negative impacts of the CAR-T boxed label warning during a recent listening session indicates the agency may be thinking about improving its dissemination of the information. 

Regulators in Europe and the US have demonstrated a commitment to providing guidance that will make decentralized and point-of-care manufacturing a reality, the CSO of GermFree, a company that provides mobile and modular cleanrooms for manufacturing advanced therapies, tells the Pink Sheet.

After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.

In this week's podcast edition of Five Must-Know Things: summer ADC dealmaking continues apace; a look at cell therapies in Japan; Pfizer’s new US digital consumer platform; Galapagos’s US point of care CAR-T study; and Novartis partners with Lindy on biologics delivery.