Spark Therapeutics to Present Updated Hemophilia B Data at the International Society on Thrombosis and Haemostasis (ISTH) 201...
June 23 2017 - 2:00AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company dedicated to challenging the inevitability of genetic
disease, announced today that Lindsey George, M.D., attending
physician in the Division of Hematology at Children's Hospital of
Philadelphia, and a clinical investigator in the Phase 1/2 clinical
trial of SPK-9001 for hemophilia B, will present updated
interim data at the International Society on Thrombosis and
Haemostasis (ISTH) 2017 Congress in Berlin.
Additionally, Katherine A. High, M.D., president and chief
scientific officer at Spark Therapeutics, will provide an overview
of gene therapy research in hemophilia, including updated interim
data on SPK-9001, during Tuesday’s “State-of-the-Art” session.
Sylvia von Mackensen, Ph.D., senior scientist at the Institute of
Medical Psychology, University Medical Centre Hamburg-Eppendof,
Germany, will present a poster on quality-of-life data of
participants in the SPK-9001 clinical trial.
Specific dates and times for the presentations are:
- “SPK-9001: Adeno-associated Virus Mediated Gene Transfer for
Haemophilia B Achieved Durable Endogenous Prophylaxis at Levels of
Activity Sufficient to Achieve Significant Mean Reduction in Annual
Bleeding and Infusions Rates in Preliminary Data from an Ongoing
Phase 1/2a Trial,” Lindsey George, M.D., on Monday, July 10, from
5:45 to 6 p.m. CET
- “Gene Therapy Replacement,” Katherine A. High, M.D., on
Tuesday, July 11, from 8 to 8:25 a.m. CET
- “Preliminary Results of SPK-9001 Gene Transfer Demonstrate
Statistical Improvements on the Health-related Quality-of-Life in
Adults with Haemophilia B Speaker,” Sylvia von Mackensen, Ph.D.,
poster session on Tuesday, July 11, from 12 to 1:15 p.m. CET
About Spark TherapeuticsSpark Therapeutics, a
fully integrated company, strives to challenge the inevitability of
genetic disease by discovering, developing, and delivering gene
therapies that address inherited retinal diseases (IRDs),
neurodegenerative diseases, as well as diseases that can be
addressed by targeting the liver. Our validated platform
successfully has delivered proof-of-concept data with
investigational gene therapies in the retina and liver. Our most
advanced investigational candidate, voretigene neparvovec, in
development for the treatment of biallelic RPE65-mediated IRD, has
received orphan designations in the U.S. and European Union, and
breakthrough therapy designation in the U.S. The pipeline also
includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two
hemophilia development programs: SPK-9001 (which also has received
both breakthrough therapy and orphan product designations by the
FDA, and access to the PRIority MEdicines (PRIME) Program by the
EMA) in a Phase 1/2 trial for hemophilia B being developed in
collaboration with Pfizer, and SPK-8011, in a Phase 1/2 trial for
hemophilia A to which Spark Therapeutics retains global
commercialization rights. To learn more about us and our growing
pipeline, visit www.sparktx.com.
Investor Relations Contact:
Ryan Asay
Ryan.asay@sparktx.com
(215) 239-6424
Media Contact:
Monique da Silva
Monique.dasilva@sparktx.com
(215) 282-7470
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