Translational research workshop

On June 25th, HSCI hosted its final Translational Research Workshop of the year moderated by John McNeish, PhD, the GlaxoSmithKline manager responsible for the HSCI-GSK collaboration. The topic, "Case Studies in Translational Medicine: Gaps and Opportunities for Academia-Industry Partnerships," was designed to help individuals from academia and industry understand what it takes to build successful partnerships to accelerate the movement of research from the lab into the market.

The first speaker, Jason Gardner, PhD, Head of Regenerative Medicine Discovery Performance Unit at GSK R&D, talked about an academic-industry partnership wherein they are bringing gene therapies to market. Similar to its relationship with HSCI, GSK joined forces with the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) to develop gene therapies against rare genetic disorders. The product from this alliance is in the pre-registration phase for the lead indication, two indications are in clinical trials and four more indications are in preclinical development. The partnership's success points to the positive future of the HSCI-GSK relationship as more of our joint discovery programs move into clinical development.

The second speaker, Julian Jenkins, PhD, VP of GSK's Center for Clinical Study Excellence, talked about the journey of the drug Eltrombopag from screening assays to a marketed product at a much higher-than-average speed. This experience highlighted the importance of good science, open and extensive academic collaborations, and solid judgment in the success of this drug against thrombocytopenia.

The final speaker was HSCI Executive Committee member Lee Rubin, PhD, who not only reviewed the applications and limitations of stem cells in the drug discovery and development process, but also shared advances from two of his screening programs against Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). The presentation sparked an engaging discussion on how best to advance his screening hits towards potential therapeutics, a process where the academic lab will have to rely on commercial partners.