The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for an investigational hemophilia A therapy being developed by Sernova, a Canadian-based clinical-stage biotechnology company.

A primary component of the experimental therapy is Sernova’s proprietary Cell Pouch™ technology that targets a patient’s blood outgrowth endothelial cells (BOECs). These cells line the interior surface of blood and lymphatic vessels, making up the endothelium membrane, to perform many critical functions associated with blood flow, organ, and tissue functioning. BOECs are removed from a patient, modified to affect the release of functioning FVIII into the bloodstream, and reimplanted via the Cell Pouch. Pre-clinical research into this approach to gene and cell therapies is ongoing.

While there are several advantages and broader therapeutic applications to employing this novel approach to treatment, scientists acknowledge that much additional study will be needed to answer key questions before the potential of such a therapy could be fully realized.

"BOECs are an excellent model for human endothelium, thus a very valuable tool to study endothelium in patients. The announcement from Sernova of using the patients’ own BOEC to produce FVIII is very exciting: it means that the FVIII protein will be processed correctly by the right cells which is likely to have major implications for function and immunogenicity," said Dr. Anna M. Randi, hematologist and professor of cardiovascular medicine and head of vascular sciences at the National Heart and Lung Institute. Randi also conducts research at the Imperial College London where she specializes in vascular biology and vascular hemostasis. "If the approach is successful, it will also open the way for application in other diseases due to dysfunction or lack of endothelial proteins. It will be important to understand how production will be calibrated, how long will the cells survive, how often they will need to be replaced – these are all open questions."

Sernova also announced that it is collaborating with University of Piemonte Orientale (UPO) in Italy with the aim of progressing this therapy from pre-clinical to clinical trials in hemophilia A patients. This effort is being directed by Antonia Follenzi MD, PhD Professor of Histology, and Cell and Gene Therapy at UPO.

“I am pleased to be working with the Sernova team on these advanced technologies for a new and safe treatment of patients with hemophilia A using a gene and cell therapy approach within the Cell Pouch,” said Follenzi. “Our goal is to advance these new technologies to further maximize the release of FVIII into the bloodstream and to extend the duration of that release for a sufficient duration of time to eliminate the need for weekly infusions of FVIII and to significantly improve the lives of people with hemophilia A.”

The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments. RPDD was established with some similar objectives as ODD but with particular focus on incentivizing research and development for new therapies for children with rare conditions.

“We are pleased with the FDA’s decision to grant these designations for our novel treatment for Hemophilia A, which uses the Cell Pouch™ in combination with cells corrected for the production of Factor VIII,” commented Cynthia Pussinen, Chief Executive Officer of Sernova. “Hemophilia A is a serious, life limiting condition and we are committed to advancing development of the program, with a hope to positively impact patients around the world who are waiting for improved treatments.”

Additional Resource

VWF Regulation in Blood Outgrowth Endothelial Cells with Dr. Christopher Ng (Wednesday Webinar, NBDF)

Source: Sernova press release dated November 27, 2024