Peer Exchange: Fixing the Potholes in Cell and Gene Therapies

Success and Challenges (Potholes) in Cell and Gene Therapy: automation, regulatory certainty, and standardizing manufacture processes such as vector constructs or Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) steps.

Standardization leading to CGT therapies becoming a standard of care. Importance to outcomes of high-quality starting materials, to reducing batch-to-batch quality, and the possibilities of allogeneic chimeric antigen receptor therapy (CAR-T) successes.

Popular new delivery vectors, and some not fully appreciated like Non-AAV parvoviruses or Herpes Simplex virus. Are we in a transition state, where there is a growing a trend away from Viral Vectors? How do we best regulate CRISPR?

Repairing nucleotide abnormalities for rare disease populations as pivotal proving grounds for transformative therapies.

Looking ahead 5-10 years for in vivo practice. Combined platform and modalities marriages, to bring CGT up to first relapse or consolidation therapies.

Optimizing analytics, including laser force cytology, with a goal to delivering adaptive manufacturing, full automation and standardized processes, products and patient outcomes.

Addressing manufacturing at scale for ATMP’s. Reducing costs through reducing batch failures and shrinking footprints, and targeting more focused critical quality attributes for potency but also patient efficacy. Reducing regulatory uncertainty in an uncertain environment to increase patient access to life saving cures.