Phoenix Nest Inc. Licenses a Sanfilippo Syndrome Gene Therapy From the University of Manchester and is Preparing for a Clinical Trial
Nov 7, 2018, 2:15pm EST
NEW YORK, Nov. 7, 2018 /PRNewswire/ -- Phoenix Nest Inc. exclusively licensed a gene therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIIC, (MPSIIIC)) from The University of Manchester through its IP commercialization arm UMI3 Ltd. MPS IIIC is a devastating neurodegenerative lysosomal storage disorder of childhood for which there is no cure or effective treatment available.
Dr. Brian Bigger (Professor of Cell and Gene Therapy, University of Manchester) states "our recent publication in Brain* on this rAAV gene therapy demonstrated preclinical evidence from behavioral and cognitive tests which provide an expectation of direct clinical benefit. The vector we are using has an established safety profile and is in widespread use in treating conditions of neurological origin. It is our expectation that this will translate into a successful treatment for this disease."
"We are excited about the potential of this gene therapy. Phoenix Nest Inc. has an orphan designation for this treatment from the EMA and is currently submitting this designation to the FDA along with the Pre-IND. We are also starting vector manufacturing in preparation for a clinical trial in the very near future," said Ms. Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest.
"Our focus on developing different treatment modalities for Sanfilippo syndrome leverages a wide array of academic partners. Our strategy can help us translate these outstanding preclinical efforts and bring them to the patients faster. This creates considerable value for all stakeholders," said Dr. Sean Ekins, CEO and Co-Founder.
"Phoenix Nest is in great admiration of the parents of children affected by Sanfilippo Syndrome. These families have taken a disease with no hope of survival to the brink of a clinical trial. These are the heroes that deserve all the credit for identifying and funding leading academics and inspiring the scientists to create a drug for this insidious disease. If not for my child, then for the next generation, no parent should have to watch their child slowly die of a genetic syndrome that can be treated," states Jill Wood. #
* Brain, 2018 Jul 1;141(7):2014-2031
# https://www.huffingtonpost.com/entry/rare-genetic-disorder-gene-therapy-multiple-sulfatase-deficiency_us_5bcddc11e4b0d38b587a8bf7
About Phoenix Nest, Inc. - Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome Co-founded by Jill Wood (a parent of a child with Sanfillipo Syndrome type C) and Dr. Sean Ekins (a scientist with over 20 years of experience in pharma R&D). The company collaborates with various academics to develop treatments for Sanfilippo Syndrome. Phoenix Nest has been the recipients of 5 NIH grants to date.
For further information, please contact Sean Ekins, Co-Founder and CEO, Phoenix Nest, 215-687-1320 sekins@phoenixnestbiotech.com
About University of Manchester/UMI3 Ltd
The University of Manchester, a member of the prestigious Russell Group, is the UK's largest single-site university with 38,600 students and is consistently ranked among the world's elite for graduate employability.
The University is also one of the country's major research institutions, rated fifth in the UK in terms of 'research power'. World class research is carried out across a diverse range of fields including cancer, advanced materials, addressing global inequalities, energy and industrial biotechnology.
UMI3 Ltd is wholly owned by the University, which has over a 30-year history of IP commercialization. For more information, please visit www.umip.com
Related Links
Phoenix Nest Biotech Inc
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Phoenix Nest Inc., Los Angeles Biomedical Research Institute (LA BioMed) and Washington University in St. Louis Announce a New NIH Grant for Sanfilippo Syndrome (MPSIIID)
Nov 7, 2018, 2:23pm EST
NEW YORK, Nov. 7, 2018 /PRNewswire/ -- The National Institute of Neurological Disorders and Stroke at the National Institutes of Health (NIH) recently awarded $5,612,276 in SBIR Phase IIB grant funding to Phoenix Nest, Inc., The Division of Medical Genetics at the Los Angeles Biomedical Research Institute (LA BioMed) and Washington University in St. Louis to develop a therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIID, (MPSIIID)). This is a devastating neurodegenerative lysosomal storage disorder of childhood for which there is no cure or effective treatment available.
Dr. Patricia Dickson (Division Chief, Genetics and Genomic Medicine, Washington University in St. Louis) states, "Dr. Tsui-Fen Chou (Assistant Professor of Pediatrics, LA BioMed), Phoenix Nest Inc, and myself have collaborated over the last four years to develop an enzyme replacement treatment for MPS IIID that shows promise in an animal model of the disease. Our strategy proposes to deliver the recombinant human alpha-N-acetylglucosamine-6-sulfatase (rhGNS) as an enzyme replacement therapy to treat patients. This grant will allow us to develop significant quantities of protein and test its safety prior to a clinical trial."
"Enzyme replacement therapy, giving patients a working enzyme to replace a mutated or missing one in the body, is a proven strategy that has been applied to several lysosomal storage diseases over the last 20 years. We are quite literally standing on the shoulders of these scientists who have led the way and demonstrated that it is possible," said Dr. Tsui-Fen Chou.
"We are excited to continue our collaboration as we develop this enzyme replacement further with the support of the NIH. Our ultimate goal is to commercialize this treatment and ensure it reaches the patients we know are out there waiting for it," stated Dr. Sean Ekins, CEO of Phoenix Nest Inc.
"Phoenix Nest Inc. was founded by Sanfilippo families to develop treatments for the different forms of Sanfilippo syndrome. These terrible illnesses cause enormous suffering for children and our families. As a parent of a child with this syndrome, I know this only too well. We are indebted to researchers like Dr. Dickson and Dr. Chou and their institutes for supporting this important work and progressing it so rapidly," stated Ms. Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest.
About Phoenix Nest, Inc. - Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome founded by and solely owned by several Sanfilippo Syndrome families. They actively collaborate with academic researchers to fund and commercialize their technologies. Phoenix Nest, Inc. also licenses assets from companies and universities that may have utility for Sanfilippo Syndrome. For more information, please visit http://www.phoenixnestbiotech.com.
For further information, please contact Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest, 347-689-2186 jwood@phoenixnestbiotech.com
About LA BioMed
LA BioMed is an incubator of innovation with a global reach and a 66-year reputation of improving and saving lives. Driven by the positive social impacts of breakthrough therapies, the Southern California-based research institute, and incubator – with over 100 principal investigators (PhDs, MDs and MD/PhDs) working on over 600 research studies, including therapies for multiple orphan diseases – is an essential element to one of the nation's top-ten biotech clusters. Find out more at www.labiomed.org.
Contact information Washington University School of Medicine in St. Louis
Judy Martin Finch, director of media relations
314-286-0105
martinju@wustl.edu
Disclaimer
Research reported in this publication was supported by the National Institute of Neurological Disorders and Stroke of the NIH under Award Number R44NS089061. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.
