March 3, 2021
Damon R. Race, President & CEO
RALEIGH, NC – GeneVentiv Therapeutics, a gene therapy company developing a safe, effective, and durable, single infusion treatment for all types of hemophilia with or without inhibitors, today announced the expansion of the Company’s Scientific Advisory Board (SAB) with the addition of four leading experts in hemophilia and gene therapy.
David Lillicrap, M.D., FRCPC, Steven Pipe, M.D., Benjamin Samelson-Jones, M.D., Ph.D. and Linda Couto, Ph.D. will join the SAB to help guide the Company’s efforts to advance a potential universal, gene therapy cure for all types of hemophilia with or without inhibitors through pre-clinical testing and into the clinic.
Concurrent with the expansion of the SAB, the Company is appointing Dr. Valder Arruda to the role of Chairman of the Scientific Advisory Board. The expertise of the SAB members in hemophilia, gene therapy and the biochemistry of coagulation will assist GeneVentiv be first to market for inhibitor patients and provide the first, single infusion therapy for hemophilia patients of all types.
“We are extremely proud to have Drs. Lillicrap, Pipe, Samelson-Jones and Couto join our world-class scientific advisory board and we are especially proud to announce Valder’s leadership role on the team” said Damon Race, President and CEO of GeneVentiv Therapeutics. “As we rapidly advance our next-generation, universal gene therapy for all types of hemophilia patients toward the clinic, the guidance from our SAB is critical to our continued success.”
David Lillicrap, M.D., FRCPC is a renowned expert on the molecular aspects of the hemostatic (coagulation) system and a Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada. He is the recipient of a Senior Canada Research Chair in Molecular Hemostasis. In 2013, he was elected to the Fellowship of the Royal Society of Canada. Dr. Lillicrap is a member of the World Federation of Hemophilia’s (WFH) Medical Advisory Board and a past Chair of the WFH’s Research Committee. He is a past Chairman of the International Society on Thrombosis and Haemostasis’ (ISTH) Scientific and Standardization Committee and is a current member of the Council of ISTH. Between 2008-2014 he served as an Associate Editor of Blood and is currently Co-Editor-in-Chief of the Journal of Thrombosis and Haemostasis. Dr. Lillicrap’s research interests are centered on molecular aspects of the hemostatic system, with a particular emphasis on the potential of molecular genetics and molecular biology to address questions relating to pathological hemostasis. Studies range from an investigation of the mechanisms regulating production of the adhesive hemostatic protein, von Willebrand Factor, to the development of strategies for gene therapies for the inherited bleeding disease, hemophilia.
“GeneVentiv’s approach to gene therapy for hemophilia is the first to target increased expression of activated Factor V in the common pathway, making it uniquely positioned to be the first potential cure for inhibitor patients, while simultaneously offering a potential cure for both hemophilia A and B,” said Dr. Lillicrap. “I look forward to working with this dedicated team of experts to shape the development of the first gene therapy that could cure hemophilia patients of all kinds.”
Steven Pipe, M.D., is a pioneering clinician-scientist recognized for his expertise in hemophilia and coagulation disorders. Dr. Pipe is a Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan, Ann Arbor, Michigan, USA. He is the medical director of the Pediatric Hemophilia and Coagulation Disorders Program and medical director of the Special Coagulation Laboratory. His clinical interests include bleeding and thrombotic disorders and congenital vascular anomalies. Dr Pipe also directs a basic research laboratory investigating coagulation factor VIII and the molecular mechanisms of hemophilia A. He was the 2015 recipient of the Leadership in Research Award from the National Hemophilia Foundation. He has served on the Board of Directors for the Hemostasis and Thrombosis Research Society, as Chair of the Board of Directors for the American Thrombosis and Hemostasis Network and currently as Chair of the Medical and Scientific Advisory Committee to the National Hemophilia Foundation.
“A greater understanding of the potential of utilizing the common pathway to restore hemostasis in hemophilia patients holds the promise of extending a potential cure to all patients by bypassing any missing upstream factors and inhibitors,” said Dr. Pipe. “I am excited to be part of GeneVentiv and the potential of a singular therapy that can treat so many different types of hemophilia patients.”
Benjamin J. Samelson-Jones, M.D., Ph.D., is an attending physician in the Division of Hematology at Children’s Hospital of Philadelphia (CHOP). He is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. He is board certified in hematology/oncology and pediatrics. He specializes in treating children with bleeding or clotting disorders, sickle cell disease, and other blood disorders. His research at CHOP helped establish the gain-of-function coagulation factor IX variant Padua as the standard transgene for gene therapy for hemophilia B. His current research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.
“I look forward to working with GeneVentiv to achieve their mission of developing a potential gene therapy cure for all hemophilia patients. With gene therapies for hemophilia A and B in the forefront, such treatment for patients with inhibitors needs to be developed,” said Dr. Samelson-Jones. “As a part of GeneVentiv’s Scientific Advisory Board, I look forward to giving these patients new hope.”
Linda Couto, Ph.D., joins GeneVentiv’s SAB as an independent consultant, following a recent position as Head of Pharmacology and Toxicology for Spark Therapeutics. Dr. Couto brings over 25 years of adeno-associated viral (AAV) vector-mediated delivery of therapeutic genes to GeneVentiv’s SAB. Dr. Couto’s pre-clinical development of AAV vectors spans multiple therapeutic areas including hematology, ophthalmology, audiology, and infectious disease. Her pre-clinical development expertise includes non-clinical pharmacology, biodistribution and toxicology. Dr. Couto has authored multiple non-clinical modules to support applications at development stages from IND to BLA and MAA, including Luxturna, the first gene therapy licensed in the US for a genetic disease.
“Advancing pre-clinical gene therapy products into the clinic and regulatory submissions are familiar territory for me,” said Dr. Couto. “I look forward to guiding GeneVentiv’s efforts to rapidly advance their lead hemophilia program to an IND and am excited about the Company’s current efforts to in-license additional programs.”
About GeneVentiv Therapeutics:
GeneVentiv Therapeutics, Inc. is developing the first effective gene therapy for Hemophilia A and Hemophilia B with or without inhibitors. Patients with inhibitors are unable to effectively respond to current therapies and gene therapies in development due to neutralizing antibodies (inhibitors) formed by the body in response to treatment with missing clotting factor. GeneVentiv’s lead gene therapy, GENV-HEM, enables a patient’s liver to produce activated clotting Factor V, a co-factor in the coagulation cascade, which allows normal clotting in response to injury, without thrombotic risk. Our therapy will be first to market for hemophilia patients with inhibitors for whom no single infusion gene therapies are currently available.