2019-10-26 22nd SAPA-NE Scientific Symposium 【 3rd Gene Therapy Symposium】 Leap Forward to Cure

October 26th, 2019, Boston, the world’s renowned center for education and innovation in the global medical field, welcomed Sino-American Pharmaceutical Professionals Association (SAPA)’s New England Chapter (SAPA-NE) members, world-renowned leaders and regulators to join a full day 22nd SAPA-NE scientific symposium “Leap Forward to Cure” at the Mark and Lisa Schwartz Auditorium, Ragon Institute of MGH, MIT and Harvard, 400 Technology Square, Cambridge.

More than 200 SAPA-NE members and friends gathered to listen to 10 invited speakers, representing academic institutions, industry leaders and Regulatory agency. The conference was opened with remarks from Dr. Qiying Hu, the SAPA-NE President. Dr. Hu shared the vision and mission of SAPA-NE, the incredible progress of this community, as well as its contribution to the success of the biotech industry in the New England area.

The morning session featured multiple lectures from academic, industry, and regulatory leaders, who shared their views on the development of gene therapy to address unmet medical needs. The keynote speech was given by Dr. Guangping Gao, Professor and Director of Horae Gene Therapy Center at UMass Medical School, cofounder of Voyager Therapeutics, and the current President of American Society of Gene and Cell Therapy. Dr. Gao, introduced the milestones in the development of gene therapy, shared recent progress from his laboratory and collaborators on recombinant adeno-associated virus (AAV) gene therapy vector research, and its application on developing a potential therapy for Canavan’s disease. Dr. Ricardo Brau, Managing Director at L.E.K. Consulting, discussed gene therapy from the commercial and business opportunity perspectives. Interesting business topics, including the comparison between curative gene therapy versus traditional chronic therapy in terms of innovative commercial models and company pipeline management were discussed. Opportunities and challenges of the current gene therapy research and development (e.g. limitations of current process development of gene therapy and CMC management at expedited clinical-trial design) were also discussed and valuable viewpoints were shared. From regulatory side, Dr. Ke Liu, Associate Director for Cell and Gene Therapy, Oncology Center of Excellence & Chief of Oncology Branch at FDA, delivered an exciting presentation on his perspectives of preclinical and clinical considerations in developing gene therapy. This is followed by a presentation delivered by Dr. Nripen Singh, Director of Biopharmaceutical Development at Voyager Therapeutics, discussing the AAV platform development for the process developments, process characterization, unit operations during CMC of gene therapy products at Voyager.

Dr. Qiying Hu, Prof. Guangoing Gao and Dr. Jason Jingxin Zhang.

Dr. Ricardo Brau

Dr. Ke Liu

Dr. Nripen Singh

Questions and Discussion

Speakers and Organizing Committees

The beautiful fall day was perfect for many old and new friends to enjoy the sunshine and lunch table discussion in the courtyard of Technology Square. The afternoon session started by Dr. Hans G. Klingemann, Vice President of Research & Development from NantKwest. Dr. Hans reported the positive results and progress in its NK cell pipeline programs for the treatment of cancer. NantKwest is an innovative clinical-stage immunotherapy company focused on harnessing the power of the innate immune system by using the natural killer cell to treat cancer, infectious diseases and inflammatory diseases. Dr. Hans shared the progress and promise of NK cell based platforms that have been bioengineered to incorporate chimeric antigen receptors (CARs) and antibody receptors to potentially further optimize targeting and potency for the disease treatment. Dr. Jeffrey Palmer, Senior Director of Pfizer shared his perspectives on the clinical trial and FDA approval landscape of gene therapy. Currently, Pfizer is focused on gene therapy treatments for diseases that have single-gene alterations. Their approach is to use recombinant adeno-associated virus (rAAV) that are designed to deliver treatment effectively to the targeted tissue. Dr. Jeffrey Palmer introduced FDA's efforts to advance the development of Gene Therapy through accelerated approval and other programs. He used case studies from Pfizer, such as hemophilia B and Duchenne muscular dystrophy (DMD) gene therapy programs to illustrate how gene therapy clinical trials were designed to meet the challenges of 1) small sample sizes; 2) fewer opportunities to pause and answer questions in an informed way; 3) some indications have no established registrational endpoints. Dr. Alasdair Milton, Vice President at Parthenon-EY discussed the commercial model perspectives of gene therapy. Dr. Alasdair Milton explored how companies can effectively deploy the capital to re-shape their gene therapy portfolios in order to take advantage of this ‘innovation surge.’ Specifically, Dr. Alasdair Milton used Spark Therapeutics as an example to demonstrate how the company has been doing a good job in improving manufacturing as well as building a patient-centric pharma-patient relationship by establishing a specialized medical sciences liaison team. One of the biggest challenges for pharma when developing gene therapy programs is to accurately document the treatment durability. Since patients tend not to go back to doctors for follow-ups after treated by gene therapy. So building a strong patient-pharma relationship is critical for treatment durability documentation.

Drs. Qiying Hu, Hans G, Klinggemann and Jason Jingxin Zhang

Dr. Jerrery Palmer