Spark Therapeutics Announces Collaboration with Leading Gene Therapy Center at University of Massachusetts Medical School
October 20 2016 - 4:30AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company seeking to transform the lives of patients with
debilitating genetic diseases by developing investigational,
potentially one-time, life-altering treatments, announced today a
multi-year research agreement with Guangping Gao, Ph.D., the
Penelope Booth Rockwell Chair in Biomedical Research, director of
the Horae Gene Therapy Center, and professor of microbiology and
physiological systems at the University of Massachusetts Medical
School. Dr. Gao will collaborate broadly with Spark Therapeutics to
identify adeno-associated virus (AAV) vectors from a proprietary
library of AAV capsids and evaluate their efficacy, with the goal
of enhancing the efficiency of gene delivery to cells in the
retina, liver and central nervous system.
“We’re excited to have selected Spark Therapeutics as a
collaborator to develop next-generation AAV vectors that deliver
genetic information to certain cell types more efficiently and to
cell types that are otherwise hard to target,” said Dr. Gao, an
internationally renowned AAV and gene therapy researcher.
Under the agreement, Spark Therapeutics has the option to an
exclusive, world-wide license for the intellectual property
developed under the research agreement.
“We are delighted to be working with Professor Gao, a long-time
leader in gene therapy and capsid development,” said Katherine A.
High, M.D., president and chief scientific officer at Spark
Therapeutics. “Strategically, these types of relationships and
agreements help us build on our research in gene therapy and
continue to advance the utility of our proven and proprietary gene
therapy platform.”
About Spark TherapeuticsSpark Therapeutics, a
fully integrated gene therapy company, is seeking to transform the
lives of patients with debilitating genetic diseases by developing
investigational, potentially one-time, life-altering treatments.
Spark Therapeutics’ validated gene therapy platform is being
applied to a range of clinical and preclinical programs addressing
serious genetic diseases, including inherited retinal diseases,
liver-mediated diseases such as hemophilia, and neurodegenerative
diseases. Spark Therapeutics’ validated platform successfully
has delivered proof-of-concept data with investigational gene
therapies in the retina and liver. Spark Therapeutics has reported
top-line results from a pivotal Phase 3 clinical trial for its most
advanced product candidate, voretigene neparvovec (formerly
referred to as SPK-RPE65), a potential treatment of a rare genetic
blinding condition. Voretigene neparvovec has received both
breakthrough therapy and orphan product designations. Spark
Therapeutics’ hemophilia franchise has two lead
assets: SPK-9001 in a Phase 1/2 trial for hemophilia B
being developed under a collaboration with Pfizer and SPK-8011, a
preclinical candidate for hemophilia A to which Spark Therapeutics
retains global commercialization rights. To learn more, please
visit www.sparktx.com.
About the University of Massachusetts Medical
School The University of Massachusetts Medical School, one
of five campuses of the University system, is comprised of the
School of Medicine, the Graduate School of Biomedical Sciences, the
Graduate School of Nursing, a thriving research enterprise and an
innovative public service initiative, Commonwealth Medicine. Its
mission is to advance the health of the people of the Commonwealth
through pioneering education, research, public service and health
care delivery with its clinical partner, UMass Memorial Health
Care. In doing so, it has built a reputation as a world-class
research institution and as a leader in primary care education. The
Medical School attracts more than $266 million annually in research
funding, placing it among the top 50 medical schools in the nation.
In 2006, UMMS’s Craig C. Mello, Ph.D., Howard Hughes Medical
Institute Investigator and the Blais University Chair in Molecular
Medicine, was awarded the Nobel Prize in Physiology or Medicine,
along with colleague Andrew Z. Fire, Ph.D., of Stanford University,
for their discoveries related to RNA interference (RNAi).
Spark Therapeutics Corporate Contacts
Stephen W. Webster, Chief Financial Officer
Daniel Faga, Chief Business Officer
(855) SPARKTX (1-855-772-7589)
Media Contact
Dan Quinn
Ten Bridge Communications
(781) 475-7974
dan@tenbridgecommunications.com
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