|[April 15, 2014]
AAVLife Raises $12 Million in Series A Financing to Advance Gene Therapy for Friedreich's Ataxia
PARIS --(Business Wire)--
AAVLife, a gene-therapy company focusing on rare diseases, today
announced that it has raised $12 million in Series A financing to
advance into clinical studies the promising work on Friedreich's ataxia
recently reported in Nature Medicine.
Versant Ventures, an international life-sciences venture-capital firm,
led the financing round with participation from Inserm Transfert
Initiative, a French seed-investment firm linked to the French National
Institute of Health and Medical Research (Inserm).
"Gene therapy is making great strides, with recent findings showing the
potential to significantly improve the lives of patients with genetic
diseases," said Thomas Woiwode Ph.D., a Venture Partner at Versant. "At
AAVLife we have assembled a world-class team focused on driving this
program into the clinic to bring true benefit to patients with
Matthieu Coutet, Managing Partner at Inserm Transfert Initiative, said,
"We are privileged to have early access to exciting research at Inserm
and see a compelling need to transition the Friedreich's ataxia program
from the laboratory to the bedside."
Friedreich's ataxia is caused by a mutation in the frataxin gene, which
results in progressive neurologic and cardiac symptoms typically first
appearing in childhood or during the teenage years. Progressive
degradation of cardiac function accounts for the majority of deaths from
this disease. There are no approved therapies.
The research paper in Nature Medicine reported the successful use
of a gene-therapy approach in a mouse model developed by deleting the
frataxin gene. The authors report that these mice develop the same
progressive degradation of cardiac function, and that delivery of a
normal frataxin gene using an adeno-associated virus (AAV) completely
restored normal cardiac function and reversed pathological heart
enlargement in mice that had already progressed to heart failure.
AAVLife has already advanced research of this gene-therapy approach into
further preclinical studies to inform decisions about dosing and route
of administration to be used in a clinical trial. The company will be
conferring with regulatory authorities about toxicity-study requirements
and clinical-trial design. The goal is to commence a clinical trial in
2015 to evaluate gene therapy for the cardiac dysfunction associated
with Friedreich's ataxia.
"Friedreich's ataxia may be a rare disorder, but it is an all too common
feature of everyday life for patients and their families," said Amber
Salzman, Ph.D., a co-founder and the Chief Executive Officer of AAVLife.
"We are moving with appropriate care but also urgency."
The founders of AAVLife have already played prominent and various role
in bringing gene therapy into clinical use:
-- Patrick Aubourg, M.D. Ph.D, Head of Neuropediatrics at Hôpital
Bicêtre Paris Sud. Dr. Aubourg led the first clinical trial to
use gene therapy in the treatment of adrenoleukodystrophy (ALD), a
genetic disorder that can result in fatal neurodegeneration. He and his
research group won the 2010 Grand Prix scientific award of the Simone
and Cino del Duca Foundation for their discoveries in the field of
genetic diseases of the central nervous system.
-- Pierre Bougnères, M.D., Ph.D., Head of Pediatric Endocrinology
at Hôpital Bicêtre Paris Sud. With Dr. Aubourg, Dr. Bougnères
developed and performed the ALD trial.
-- Ronald Crystal, M.D., Chairman of Genetic Medicine and
Professor of Internal Medicine at Weill Cornell Medical College, New York.
Dr. Crystal leads the research group that first used a recombinant virus
as a vehicle for in vivo gene therapy. The group has carried out
human trials of gene therapy for cystic fibrosis, cardiac ischemia,
cancer, and central-nervous-system disorders. This year the
peer-reviewed journal Human Gene Therapy presented Dr. Crystal
its Pioneer Award in recognition of his seminal work on adenoviral
-- Hélène Puccio, Ph.D., Head of a research team at the Institute
of Genetics and Molecular and Cellular Biology, University of
Strasbourg, France. Dr. Puccio has concentrated her research on
the pathophysiological mechanisms involved in ataxias such as
Friedreich's ataxia. She is the lead author on the paper in Nature
-- Amber Salzman, Ph.D., Chief Executive Officer. Dr.
Salzman, a mathematician by training, served as a member of the R&D
executive team at GlaxoSmtihKline, where she planned and managed
drug-development projects and clinical trials comprising over 30,000
patients worldwide. Subsequently, she led Cardiokine Inc. as CEO prior
to its acquisition by Cornerstone Therapeutics. She is the president of
the Stop ALD Foundation, a patient-advocacy group driving forward
improved ALD therapy. She played a key role in bringing about the
initial ALD gene-therapy trial.
About Friedreich's ataxia
Friedreich's ataxia affects 10,000 to 20,000 people in the United States
and Europe. Symptoms usually appear early in life, between ages 5 and
15, though they may be delayed until adulthood. An early symptom is loss
of control of body movements (ataxia), often starting with difficulty in
walking, then spreading to the upper limbs and trunk. The nervous-system
damage is often accompanied by heart damage. Cardiac symptoms may
include enlargement of the heart, fibrosis of heart tissue, and
heart-rhythm abnormalities. Friedreich's ataxia is caused by a mutation
in a gene termed FXN. The mutation limits production of the
protein encoded by FXN, called frataxin, which is essential to
normal function of mitochondria, the "energy factories" of the cell. The
disorder is autosomal recessive, meaning that it arises when a person
has inherited two copies of the mutated gene, one from each parent.
AAVLife, registered in Paris, is a privately held company dedicated to
advancing gene therapy for rare diseases. Further information is
available at www.aavlife.com.
Versant Ventures is a leading venture capital firm that specializes in
investment in innovative, groundbreaking bio-pharmaceuticals, medical
devices, and other life science opportunity. Founded in 1999, the firm
consists of an experienced team committed to helping entrepreneurs build
successful companies that impact healthcare and improve quality of life.
In addition to an industry-leading biotech-focused team in Basel,
Switzerland and the San Francisco Bay Area, Versant has a presence in
two other geographic locations of Southern California and Minnesota.
Further information is available at www.versantventures.com.
About Inserm Transfert Initiative
Inserm Transfert Initiative is a life-sciences seed-investment firm
based in Paris, France, linked to the French National Institute of
Health and Medical Research (Inserm). Inserm Transfert Initiative
focuses its investments in companies coming from public research
laboratories. Inserm Transfert Initiative managed €39M and is supported
by Inserm, BPI France (through the FNA funds), top-tier pharmaceutical
companies and an insurance company. Inserm Transfert Initiative has
invested in over 20 companies since 2001. For more information please
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