Spark Therapeutics Enters into Licensing Agreement with Genethon
September 20 2017 - 7:30AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy
company dedicated to challenging the inevitability of genetic
disease, today announced it has entered into a licensing agreement
with Genethon, a non-profit research and development organization
dedicated to the development of gene therapies for orphan genetic
diseases from research to clinical validation, for the development
and commercialization of an adeno-associated viral (AAV) gene
therapy targeting the liver to address a rare genetic disease.
Spark Therapeutics has the option to develop and
commercialize any gene therapy that results from the licensing
agreement. Under the terms of the agreement, Genethon will
collaborate with Spark Therapeutics on further preclinical research
activities. The specific genetic target and financial terms of the
agreement have not been disclosed.
“Capitalizing on our expertise and success to date in
liver-directed AAV gene therapy, we look forward to collaborating
with Genethon to develop a potential new gene therapy for a
debilitating genetic disease,” said Katherine A. High, M.D.,
president and chief scientific officer of Spark Therapeutics. “We
believe our agreement with Genethon expands our proven and
proprietary gene therapy platform and helps deliver on our mission
to challenge the inevitability of genetic disease.”
“Genethon is pleased to collaborate with Spark Therapeutics, a
strong partner for the development of new gene therapies,” said
Frédéric Revah, chief executive officer of Genethon. “It is a
recognition of the quality of translational research performed by
Genethon’s team with the objective of delivering effective
therapies to patients affected with rare diseases.”
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, such as hemophilia. Spark
Therapeutics has ongoing clinical trials investigating gene
therapies in hemophilia A and B. SPK-8011 is in an
ongoing, dose-escalation Phase 1/2 clinical trial as a potential
one-time therapy for hemophilia A. The company retains full global
commercialization rights to SPK-8011. SPK-9001 is in a Phase
1/2 clinical trial for hemophilia B and is being developed in
collaboration with Pfizer. It has received both breakthrough
therapy and orphan product designations from the Food and Drug
Administration (FDA), and access to the PRIority MEdicines
(PRIME) Program from the European Medicines Agency (EMA). Our most
advanced investigational candidate, with proposed trade name
LUXTURNA™ (voretigene neparvovec), is currently under Priority
Review with FDA for the treatment of patients with vision loss
due to confirmed biallelic RPE65-mediated IRD and has been
designated as a drug for a rare pediatric disease. The Marketing
Authorization Application for LUXTURNA has been validated by EMA
for the treatment of patients with vision loss due to Leber
congenital amaurosis (LCA) or retinitis pigmentosa (RP) IRDs caused
by confirmed biallelic RPE65 mutations. LUXTURNA has received
breakthrough therapy and orphan product designations
from FDA and orphan product designations from EMA. The
pipeline also includes SPK-7001 in an ongoing Phase 1/2
clinical trial for choroideremia. For more information, visit
www.sparktx.com, and follow us on Twitter and LinkedIn.
Spark Cautionary Note on Forward-looking
StatementsThis release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995, including statements regarding the company's
SPK-FIX program. Any forward-looking statements are based on
management's current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in,
or implied by, such forward-looking statements. For example, our
agreement with Genethon may not produce product candidates that
warrant further development and may not advance our gene therapy
platform. For a discussion of other risks and uncertainties, and
other important factors, any of which could cause our actual
results to differ from those contained in the forward-looking
statements, see the "Risk Factors" section, as well as discussions
of potential risks, uncertainties and other important factors, in
our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q
and other filings we make with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Spark undertakes no duty to update this
information unless required by law.
About GenethonCreated by the AFM-Telethon, the
French Muscular Dystrophy Association (AFM), Genethon, located in
Evry, France, is a non-profit R&D organization dedicated to the
development of biotherapies for orphan genetic diseases, from the
research to clinical validation. Genethon is specialized in the
discovery and development of gene therapy drugs and has multiple
ongoing programs at clinical, preclinical and research stages for
neuromuscular, blood, immune system, liver and eye diseases.
Investor Contact: |
|
Media Contact: |
Ryan
Asay
Ryan.asay@sparktx.com (215) 239-6424 |
|
Monique da
SilvaMonique.dasilva@sparktx.com (215) 282-7470 |
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