Synageva BioPharma Corp.
(“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today announced that three abstracts have been selected for oral presentation at the upcoming LDN WORLD Symposium being held February 8-10th in San Diego, California. Presentations include a review of the interim Phase I/II clinical data for SBC-102 in adults with late onset LAL Deficiency and a review of preclinical data for SBC-103, a recombinant enzyme replacement for Sanfilippo B Syndrome. In addition, an academic group from Yorkhill Hospital in the UK will be presenting an important breakthrough in diagnostic testing for Synageva’s lead indication, LAL Deficiency. The titles, dates and times for the oral presentations are:
About Synageva’s Lead Program
- Dr. Gregory Enns from Stanford University presents “Initial Human Experience with SBC-102, a Recombinant Enzyme Replacement Therapy in Adults with Lysosomal Acid Lipase Deficiency,” Friday, February 10, 2:30 PM (PST).
- Dr. John Hamilton from Yorkhill Hospital, Glasgow, Scotland, United Kingdom presents “A New Method for the Measurement of Lysosomal Acid Lipase in Dried Blood Spots Using the Inhibitor Lalistat 2,” Thursday, February 9, 2:00 PM (PST).
- Dr. Anthony Quinn from Synageva BioPharma presents “SBC-103, a Recombinant Enzyme Replacement Therapy, Demonstrates Potential for the Treatment of Sanfilippo Type B Syndrome,” Wednesday, February 8, 3:45 PM (PST).
is being developed as an enzyme replacement for Lysosomal Acid Lipase (LAL) Deficiency, a lysosomal storage disorder (LSD), and is a recombinant form of the human LAL enzyme. SBC-102 is currently being evaluated in global clinical trials and has been granted orphan designations by the U.S. Food and Drug Administration (“FDA”) and the European Medicines Agency. Additionally, SBC-102 received fast track designation by the FDA.
About LAL Deficiency
Lysosomal Acid Lipase (LAL) Deficiency
is a rare, autosomal recessive lysosomal storage disorder (LSD) that is caused by a marked decrease of the lysosomal acid lipase enzyme. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In these patients, the buildup of fatty material in the liver, spleen and blood vessel walls leads to complications resulting in significant morbidity and mortality. Early onset LAL Deficiency, sometimes called Wolman Disease, affects infants in the first year of life and is characterized by growth failure, malabsorption, steatorrhea and hepatomegaly and is rapidly fatal, usually within the first year of life.
About the Lysosomal Disease Network WORLD Symposium
The Lysosomal Disease Network (LDN) WORLD Symposium is an ACCME-accredited symposium which includes lectures and poster presentations on basic, translational and clinical research for lysosomal storage disorders. The goal of the meeting is to provide an interdisciplinary forum to explore and discuss specific areas of research and clinical interest related to lysosomal diseases.