EAGAN, Minn., Jan. 12, 2021 /PRNewswire/ -- Recombinetics
Inc., a leading gene editing company with platform technology
applied to biomedicine and animal agriculture, today announced a
research collaboration with AstraZeneca(LSE/STO/Nasdaq: AZN)
in a first of its kind study to inform the knowledge base of
Neurofibromatosis Type 1 (NF1).
NF1 is a pediatric disease that affects 1 in 3,000 births. NF1
patients suffer from a variety of manifestations including learning
disabilities, scoliosis, and are predisposed to tumor development.
In April 2020, selumetinib became the
first FDA approved therapy for NF1 patients for the treatment of
symptomatic, inoperable plexiform neurofibromas.
Surrogen, a subsidiary of Recombinetics Inc., has developed
several large animal models of neurofibromatoses that exhibit the
clinical features seen in patients with these diseases. In
particular, Surrogen's NF1 minipig displays café-au-lait macules, a
diagnostic marker of NF1. In addition, the model also
develops both optic pathway glioma and cutaneous neurofibromas,
both of which are manifestations of the disease that have been
challenging to study preclinically, until now.
Surrogen's NF1 minipig platform presents an exceptional
opportunity to better understand this disease and pilot much needed
treatments and cures to the millions of people throughout the world
suffering from this disease.
Together, AstraZeneca and Recombinetics Inc. will now undertake
a study to assess selumetinib for the treatment of cutaneous
neurofibromas, using Surrogen's NF1 preclinical platform. The
results of this study will provide key insights into whether
selumetinib can also be used as an effective treatment for
cutaneous neurofibromas, which occur in nearly all NF1
patients.
"Bringing new therapies to the clinic that are poised to
have a meaningful impact on patient lives is the most important
thing we can do," says Dr. Adrienne
Watson, Recombinetics Inc. Vice President of Research and
Development, "Utilizing a preclinical model that most closely
resembles the patient population is critical to these efforts."
According to Recombinetics' CEO, Mark
Platt, "Dr. Watson and her team have shown incredible skill,
dedication, and focus in bringing this powerful genetic model to
bear in the fight to treat and cure NF1. It is an honor to
partner with AstraZeneca to combine our efforts and make a
difference for NF1 patients and their families."
About Recombinetics
Founded in 2008, Recombinetics Inc. is a recognized global
leader in the development, deployment, and commercialization of
genetically engineered animals. Its four subsidiaries,
Regenevida, Surrogen, Makana, and Acceligen, have delivered
hundreds of animals to enable drug, device and therapeutic
discovery, generate transplantable cells, tissues and organs, and
provide improved health, well-being and productivity of
agricultural animals.
Contact:
Nikki Rockstroh
nikki.rockstroh@recombinetics.com
https://recombinetics.com/
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SOURCE Recombinetics, Inc.