By a News Reporter-Staff News Editor at Gene Therapy Weekly - Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that the Company has received notice from the U.S. Food and Drug Administration (FDA) of special regulatory designations for three of the Company's clinical programs (see also Sangamo Therapeutics, Inc.).
Rare Pediatric Disease (RPD) designation for SB-913 in vivo genome editing treatment for Mucopolysaccharidosis Type II (MPS II). RPD provides incentives to develop drugs for the treatment of rare diseases primarily affecting children ages 18 years or younger. In addition, a sponsor who receives a new drug approval for a rare pediatric disease may be eligible to receive a priority review voucher for a subsequent marketing application for a different product. The voucher may be sold or transferred.
SB-913 has already received Orphan Drug designation from the FDA. FDA has cleared an Investigational New Drug Application (IND) for this program, and a Phase 1/2 clinical trial evaluating SB-913 in adults with MPS II is open and screening subjects for enrollment.
Orphan Drug designation for SB-525 cDNA gene therapy for Hemophilia A. Orphan Drug designations are granted to drugs and biologics intended to treat rare diseases with a patient population less than 200,000 in the U.S. The designation provides incentives to advance development and commercialization of rare disease drugs.
FDA has cleared an IND for this program, and a Phase 1/2 clinical trial evaluating SB-525 in adults with Hemophilia A is expected to be opened and to begin screening subjects for enrollment later this quarter.
Fast Track designation for SB-FIX in vivo genome editing treatment for Hemophilia B. Fast track is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Once a drug receives Fast Track designation, early and frequent communication with the FDA is encouraged throughout the development and review process, often leading to earlier drug approval and access by patients.
The SB-FIX program has already received Orphan Drug designation from the FDA. FDA has cleared an IND for this program, and a Phase 1/2 clinical trial evaluating SB-FIX in adults with Hemophilia B is open and screening subjects for enrollment.
"Our four early clinical programs are focused on rare diseases for which new treatments are sorely needed, and we are gratified to work closely with the FDA as we advance these novel genomic therapies in human studies," said Dr. Sandy Macrae, CEO of Sangamo.
Sangamo's fourth lead clinical program, SB-318 in vivo genome editing treatment for MPS I, has already received Orphan Drug and RPD designations. FDA has cleared an IND for this program, and a Phase 1/2 clinical trial evaluating SB-318 in adults with MPS I is open and screening subjects for enrollment.
Sangamo's In Vivo Genome Editing Approach
Sangamo's ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy. Ultimately, the target population for these programs will include pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.
Keywords for this news article include: Biotechnology, Genetics, Hematology, Pediatrics, FDA Actions, Gene Therapy, Hemophilia A, Bioengineering, Clinical Research, Drugs and Therapies, Diagnostics and Screening, Sangamo Therapeutics Inc., Clinical Trials and Studies, Rare Diseases and Conditions, Government Agencies Offices and Entities.
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