Cellular & Genetic

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

Beyond the cost savings of biosimilars, the true evolution in wet AMD treatment is happening by companies developing next-generation therapies that aim to reduce injection burden, introduce novel mechanisms, and potentially alter disease progression.

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.

Productive talks with the US FDA after securing Regenerative Medicine Advanced Therapy designation haves speeded up development of bemdaneprocel, which is about to go into a registrational trial to see if it can slow down or even stop progression of the neurodegenerative disease.

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.

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.

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.   

“Proper medicine driven by data” is how C4X Discovery Holdings’ CEO Clive Dix sees the future now that his immuno-inflammation focused company has been able to identify IBD genetic response signatures in patients treated with vedolizumab.

Stakeholders looking to gain deeper insights into potential cell and gene therapy candidates and optimize R&D strategies can employ several strategies to inform portfolio management decisions.  

President Joe Biden signed an executive order the White House said represents the most comprehensive executive action to date on improving women’s health. The move follows the president’s call during his State of the Union for Congress to invest $12bn in women’s health research.