Spark Therapeutics Further Expands Gene Therapy Expertise with Appointment of Federico Mingozzi, Ph.D., as Chief Scientific O...
September 25 2017 - 7:30AM
Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene
therapy company dedicated to challenging the inevitability of
genetic disease, today announced the appointment of Federico
Mingozzi, Ph.D., as chief scientific officer. He will report to
Katherine A. High, M.D., who will continue as the company’s
president and head of R&D. Dr. High will also continue to serve
on the board of directors.
“We are excited to have Federico join and expand our exceptional
scientific team at Spark Therapeutics. He brings a wealth of
experience in research and early-stage clinical investigation of
gene therapies for rare inherited diseases to Spark Therapeutics,”
said Dr. High. “His deep understanding of cutting-edge vector
design and human immune responses to adeno-associated viral (AAV)
vectors further bolsters our ability to deliver on our growing
pipeline of clinical programs and platform capabilities.”
Dr. Mingozzi joins Spark Therapeutics from the French National
Institute of Health and Medical Research (INSERM) and Genethon, a
leading French nonprofit R&D organization focused on rare
diseases, where he led the development of liver-targeted gene
therapies for inherited diseases such as Crigler-Najjar syndrome,
and worked on the characterization of immunity and tolerance in
gene transfer. He serves as faculty at the Pierre and Marie Curie
University in Paris and Universitat Autonoma de Barcelona,
Spain.
Dr. Mingozzi has had a distinguished career in gene therapy
research, including earlier as director of translational research,
Center for Cellular and Molecular Therapeutics, at Children’s
Hospital of Philadelphia, where he was involved in the development
of gene therapies based on the AAV vector platform.
“I am thrilled to work with Spark Therapeutics’ team to help
advance gene therapy research programs directed at targets in the
retina, liver and central nervous system,” said Dr. Mingozzi.
“Spark Therapeutics is an established leader in the gene therapy
industry. I believe it is one of the places where true innovation
is breaking down barriers, and the company has the potential to
change the lives of people with rare genetic diseases.”
Throughout his career, Dr. Mingozzi has contributed to more than
90 publications, including seminal findings in the field of AAV
gene therapy. He has a Ph.D. in biochemistry and molecular biology
from the University of Ferrara, Italy and an MBA from Drexel
University in Philadelphia.
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.
Investor Contact:
Ryan Asay
Ryan.asay@sparktx.com
(215) 239-6424
Media Contact: Monique da
SilvaMonique.dasilva@sparktx.com(215) 282-7470
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