Orphan Drugs Asia Live: Developing cell therapies for rare diseases

In Orphan Drug Congress, Orphan Drugs Live by rachel

Thomas Lahlah, shares Cytonet’s experiences in developing therapies for rare diseases.

The Cytonet Group is an international biotechnology company spun out of Roche in 2000 with locations in Weinheim and Heidelberg in Germany and in Durham, North Carolina in the U.S. The company develops, produces and markets cell therapeutic products, including human liver cells.  Cytonet’s goal is to  provide alternatives to existing therapies for many diseases with a particular emphasis on liver diseases.   Cytonet is a pioneer and leader in the field of regenerative medicine and through the development of novel approaches to treatment, they are seeking to help shape the future of medicine.

Cytonet’s liver cell therapy leverages on the regenerative power of the liver to recreate missing biological functions.  It basically involves the therapeutic application of mature, cryopreserved hepatocytes harvested from non-transplantable donor organs.

Based on the results of clinical data:

  • Liver cell therapy can be considered safe in the target patient population; no specific or unexpected risks have been identified
  • Primary efficacy analysis shows significant prolongation of time to hyperammonaemic events for treated patients vs. controls
  • Incidence rates of hyperammonaemic events are much lower in treated patients vs. control. This holds true for all severity grades of hyperammonaemic events, especially severe and very severe events

Thomas then went on to share the challenges they have faced as a biotech company while developing innovative therapies for rare diseases. The one major challenge that he stressed that biotech companies should overcome is to manage resources and capacities well.