GeneVentiv Announces Appointment to Scientific Advisory Board

January 12, 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 appointment of Dr. Valder Arruda to the Scientific Advisory Board (SAB).  GeneVentiv is assembling a team of renowned hemophilia and gene therapy experts from, research, academic and clinical professions to advise on the development of the Company’s universal cure for all types of hemophilia with or without inhibitors.  Dr. Arruda will advise GeneVentiv on scientific and clinical programs including, in-vivo models, assay development, validation, and clinical study design.

“We are excited to be building a Scientific Advisory Board composed of experts in hemophilia and gene therapy that will guide the development of GENV-HEM, our lead hemophilia gene therapy program,” said Race. “Valder brings decades of experience in gene therapy and a deep clinical understanding of hemophilia and inhibitors to our development program.  Our gene therapy will be first to market for inhibitor patients.”

Valder Arruda, M.D, Ph.D., is a researcher in the Division of Hematology at The Children’s Hospital of Philadelphia and Associate Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.  Valder’s research focus is on the development of gene-based strategies for the treatment of bleeding and thrombotic diseases.  His current projects are focused on translational research studies on the efficacy and safety of adeno-associated virus (AAV) vectors to skeletal muscle or liver of life models with severe hemophilia. His additional studies include AAV encoding anticoagulant protein C (PC) for severe inherited PC deficiency. Valder’s laboratory is also focused on the development of transgene (FVIII, FIX and PC) with enhanced biological activity that allow optimization of gene therapy strategies such as the characterization of FIX-Pauda for hemophilia B. He is also identifying biological factors that modulate AAV vector transduction and the risk of inadvertent germline transmission in animal models that form the basis for safety studies in gene therapy by intravascular delivery of AAV vectors.

“Dr. Arruda will be influential in constituting a Scientific Advisory Board with expertise from multiple fields including hematology, hepatology, gene therapy, and immunology to provide strategic guidance and direction for the scientific and clinical development of our gene therapy for hemophilia,” said Audrey Sharbaugh, VP Clinical Development at GeneVentiv.

“Complex science and its practical application can be best understood by assembling a united team composed of clinicians, academics and researchers each drawing on expertise in a complimentary field,” said Dr. Arruda.

About GeneVentiv Therapeutics:

GeneVentiv Therapeutics is developing the first effective gene therapy for Hemophilia A and Hemophilia B with or without inhibitors.  Patients with inhibitors are unable to 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, 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.