The FDA grants orphan designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval. The priority review voucher may be redeemed, transferred, or sold.
“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
About Sernova’s Hemophilia A Cell Pouch System Program
Sernova’s Hemophilia A program combines the Sernova Cell Pouch™ with a patient’s own cells and will not require the use of immunosuppression medications. This therapy is intended to replace Factor VIII (FVIII) - an essential blood-clotting protein that is deficient or absent in patients with Hemophilia A; this is accomplished by correcting the patient’s own Blood Outgrowth Endothelial Cells (BOECs) and subsequently returning them to the patient via the Cell Pouch™. These modified cells function to release FVIII into the bloodstream, restoring the patient’s ability for clotting during periods of bleeding.
Collaboration with the
The company is pleased to announce a collaboration with the
The purpose of the new collaboration is to optimize the technology using lentiviral vectors to drive the expression of FVIII transgene under the control of novel promoters into BOECs of Hemophilia patients to achieve optimal sustained production of FVIII using an optimized cell dose within the Cell Pouch in an animal model of Hemophilia A. The overall goal of the collaboration is to develop a product combination along with pre-clinical results that support advancing into clinical trials in patients with Hemophilia A.
Professor Follenzi stated, “I am pleased to be working with the
BENEFITS OF ODD AND RPDD
Combined benefits of these designations include exclusive marketing rights for a seven-year period, after marketing approval, a 25% federal tax credit for clinical research expenses incurred in the
Furthermore, once the therapy is approved for marketing, it cannot be copied and sold in the
ABOUT HEMOPHILIA A
Hemophilia encompasses a group of inherited disorders that alter blood coagulation. Classical Hemophilia, also known as Hemophilia A, is a hereditary hemorrhagic disorder resulting from a congenital deficit of FVIII that manifests as protracted and excessive bleeding either spontaneously or secondary to trauma. 1 Hemophilia A is the most common form of Hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe Hemophilia A occurs in about 60% of cases where the deficiency of FVIII is less than 1% of normal blood concentration. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene.
According to the
ABOUT
FOR FURTHER INFORMATION, PLEASE CONTACT:
Corporate: | Investors: | Media: |
VP, Investor Relations | ||
cdavis@lifesciadvisors.com | hholmquist@lifescicomms.com | |
christopher.barnes@sernova.com | Tel: 212-915-2577 | Tel: 619-723-4326 |
Tel: 519-902-7923 | ||
www.sernova.com | ||
FORWARD-LOOKING INFORMATION
This release contains statements that, to the extent they are not recitations of historical facts, may constitute “forward-looking statements” that involve various risks, uncertainties, and assumptions, including, without limitation, statements regarding the prospects, plans, and objectives of the company. Wherever possible, but not always, words such as "expects", "plans", "anticipates", "believes", "intends", "estimates", "projects", "potential for" and similar expressions, or that events or conditions "will", "would", "may", "could" or "should" occur are used to identify forward-looking statements. These statements reflect management’s beliefs with respect to future events and are based on information currently available to management on the date such statements were made. Many factors could cause Sernova’s actual results, performances or achievements to not be as anticipated, estimated or intended or to differ materially from those expressed or implied by the forward-looking statements contained in this news release. Such factors could include, but are not limited to, the company’s ability to secure additional financing and licensing arrangements on reasonable terms, or at all; ability to conduct all required preclinical and clinical studies for the company’s Cell Pouch System and or related technologies, including the timing and results of those trials; ability to obtain all necessary regulatory approvals, or on a timely basis; ability to in-license additional complementary technologies; ability to execute its business strategy and successfully compete in the market; and the inherent risks associated with the development of biotechnology combination products generally. Many of the factors are beyond our control, including those caused by, related to, or impacted by the novel coronavirus pandemic. Investors should consult the company’s quarterly and annual filings available on www.sedarplus.ca for additional information on risks and uncertainties relating to the forward-looking statements.
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