Recombinetics Inc. Announces Neurofibromatosis Type 1 Research Collaboration with AstraZeneca


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.

Nikki Rockstroh

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SOURCE Recombinetics, Inc.

Company Codes: NYSE:AZN

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