Peter Marks, MD, PhD

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.

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

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

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

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?

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.

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.

The panel highlights the need for continued research, collaboration between stakeholders, and a strategic approach to leveraging mRNA's capabilities to address unmet clinical needs.

The key to advancing mRNA therapeutics lies in developing more efficient and versatile delivery systems that can overcome challenges around stability, targeting, and safety.

The discussion covers the challenges of optimizing mRNA therapies, including the diverse behaviors of mRNA in biological systems, as well as the opportunities presented by emerging mRNA technologies like circular and self-amplifying RNAs, and the role of regulators in ensuring quality control of these novel products.

The discussion explores how the principles of Operation Warp Speed can be applied to expedite rare disease therapeutic development through a new FDA pilot program aimed at streamlining the drug development process.

The panel discusses the rapid development of mRNA vaccines, the opportunities for mRNA technology to revolutionize drug discovery and gene therapy, and the need for regulatory collaboration to enable efficient review and access to these innovative treatments.

Experts discuss the critical need for cultivating public trust as mRNA technology makes promising advancements in treating a range of diseases.

The panelists discuss the advantages of rapid development, versatile applications, and precise dosing, and how they combine to signal a promising future for mRNA therapeutics.