Regenerative Medicine

Nonprofits are finding new ways to address market gaps and develop treatments for rare diseases with little commercial attraction.

The Centre for Commercialization of Regenerative Medicine has spent over a decade crafting a cell and gene therapy ecosystem in Canada. Now it is replicating the model abroad.

Artiva Biotherapeutics is hoping its NK cell therapy approach, which uses un-engineered cells, will prove effective in both oncologic and autoimmune indications and safe enough to be given in the community setting.

Circular RNA has several advantages, but the field is young. Scandinavian startup Circio Holdings believes its version of the technology will prove the most robust.

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.