Recombinetics Inc. Announces Neurofibromatosis Type 1 Research Collaboration with AstraZeneca

Rhea-AI Summary

Recombinetics Inc. announced a collaboration with AstraZeneca to study the treatment of cutaneous neurofibromas associated with Neurofibromatosis Type 1 (NF1) using preclinical models. NF1 affects 1 in 3,000 births, leading to learning disabilities and tumor predisposition. Selumetinib, the first FDA-approved therapy for NF1, will be evaluated in this research. Surrogen's NF1 minipig model mimics the human disease, providing a unique opportunity for treatment exploration. The study aims to enhance understanding and potential therapies for NF1 patients globally.

Positive

• Collaboration with AstraZeneca to study selumetinib for NF1 treatment.
• Utilization of Surrogen's NF1 minipig model for preclinical research.
• Potential to develop effective therapies for cutaneous neurofibromas, a common condition in NF1 patients.

Negative

• None.

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/

View original content:http://www.prnewswire.com/news-releases/recombinetics-inc--announces-neurofibromatosis-type-1-research-collaboration-with-astrazeneca-301205934.html

SOURCE Recombinetics, Inc.

FAQ

What is the collaboration between Recombinetics and AstraZeneca about?

The collaboration focuses on studying the treatment of cutaneous neurofibromas in Neurofibromatosis Type 1 using selumetinib.

What is Neurofibromatosis Type 1 and its prevalence?

Neurofibromatosis Type 1 affects 1 in 3,000 births and is characterized by various symptoms including learning disabilities and tumor predisposition.

What is the significance of selumetinib in this study?

Selumetinib is the first FDA-approved therapy for NF1, and the study aims to assess its effectiveness against cutaneous neurofibromas.

What unique model is being used for this research?

The research utilizes Surrogen's NF1 minipig model, which exhibits clinical features similar to human NF1 patients.

What impact does this research have for NF1 patients?

The research aims to better understand NF1 and potentially develop new treatments for millions of affected individuals worldwide.