RCS - Viralgen Vector Core - Viralgen & Elpida Partner for SPG50 & CMT4J Trials

Bayer AGAnnouncement

28/09/2023 13:00

For best results when printing this announcement, please click on link below:
http://newsfile.refinitiv.com/getnewsfile/v1/story?guid=urn:newsml:reuters.com:20230928:nRSb9871Na&default-theme=true

RNS Number : 9871N  Viralgen Vector Core  28 September 2023

 

 

 

 

Viralgen Vector Core and Elpida Therapeutics partner to manufacture gene
therapy medicines for anticipated Spastic Paraplegia 50 (SPG50) and
Charcot-Marie-Tooth disease type 4J (CMT4J) clinical trials

 

Trial participants with Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth
disease type 4J (CMT4J) could benefit from this joint effort to manufacture
new gene therapies that utilize adeno-associated virus serotype 9 (AAV9) to
deliver corrected genes

 

Sept 19, San Diego, California. Viralgen Vector Core (Viralgen) and Elpida
Therapeutics (Elpida Tx) have partnered to manufacture gene therapies for use
in clinical trials sponsored by Elpida Tx involving patients living with SPG50
or CMT4J. It is anticipated that these trials will take place at various sites
in North America and Europe and will explore potential safety and efficacy of
the new treatments to be manufactured, as well as potential quality of life
improvements.

 

SPG50 is a rare disease caused by mutations in the adaptor protein complex-4
mu 1 subunit (AP4M1), which is a protein responsible for intracellular vesicle
trafficking, specifically the sorting and transport of membrane proteins
within cells. The disease symptoms of children with SPG50 begin with low
muscle tone, microcephaly (small head) and epilepsy, then progress to affect
the entire body, resulting in intellectual disabilities and
movement/neurological abnormalities. This rare disease is known to affect only
around 90 people worldwide.(1)

 

Charcot-Marie-Tooth disease type 4J (CMT4J) is an autosomal recessive,
potentially severe form of CMT disease caused by mutations of the lipid
phosphatase FIG4, a protein involved in the regulation of phosphoinositide
lipids and membrane trafficking within cells. The same CMT4J genotype can
present symptoms ranging from mild clinical signs to severe disability.(2)

 

The partnership between Viralgen and Elpida Tx is expected to enable the
efficient manufacture of the adeno-associated vector serotype 9 (AAV9) based
therapies for use in Elpida Tx's anticipated multi-site clinical trials.
Viralgen will manufacture these therapies using its production process with
the Pro10(TM) cell line, which is exclusively licensed from AskBio and has
been found to increase scalability, performance, and yield of AAV
therapies.(3)

Terry Pirovolakis, Founder and CEO of Elpida Tx, remarks that "Viralgen is
truly an amazing partner, demonstrating their genuine commitment to advance
potential therapies for impacted patients. We feel so grateful and honored to
be working with such an incredible partner."

 

About Elpida Therapeutics

 

Elpida Tx' s mission aims to address the current significant unmet medical
needs of patients with ultra-rare diseases. Through leveraging scientific
advancements and the now well-established safety and understanding of certain
gene therapies, Elpida Tx aims to put cures in reach of families and children
who desperately and urgently need them. Elpida Tx's business model focuses on
partnerships that promote efficiency and the chance to treat a greater number
of patients, while being self-sustaining and replicable. Elpida Tx
specifically focuses on advancing programs that traditional biotech companies
find difficult to bring through to completion. The company pipeline includes
programs that were deprioritized by biotech companies or that did not receive
biotech investment to advance due to small patient populations, but which have
excellent scientific data on which to build a program and springboard the
science and opportunity. Elpida Tx plans to incorporate three more CNS
programs into its initial program pipeline within the next six months through
an application process designed for academic institutions and foundations.

 

About Viralgen Vector Core

Viralgen is a Contract Development and Manufacturing Organization (CDMO)
founded in 2017 and exists as an independently operated subsidiary of AskBio,
which is wholly owned and independently operated as a subsidiary of Bayer AG.
As a manufacturer of Current Good Manufacturing Practice (cGMP) certified AAV,
Viralgen offers the Pro10™ based suspension manufacturing platform, a
technology licensed from AskBio and developed by Chief Technical Officer Josh
Grieger, PhD, and Co-Founder R. Jude Samulski, PhD, at University of North
Carolina.  The Pro10™ platform has been found to increase scalability,
performance, and yield of AAV therapies (3). Located in Spain, in the Gipuzkoa
Science and Technology Park, Viralgen produces AAV gene therapy treatments for
pharmaceutical and biotech companies with the aim of accelerating the delivery
of new treatments that may improve patients' lives.

 

The company's clinical facilities have four cGMP manufacturing suites, with
250-liter and 500-liter bioreactors. In 2020, Viralgen expanded within the
Scientific Park by constructing a new building with three modules for
large-scale commercial manufacturing. Each module of the state-of-the-art
space includes three cGMP suites with a manufacturing capacity of >2,000
liters. The first module, which includes a suite dedicated to fully automated
fill and finish operations, has received cGMP certification by the Spanish
Agency for Medicines and Medical Devices (AEMPS) as part of the EMA network.
For more information, visit viralgenvc.com.

 

References:

(1) J Clin Invest. 2023 May 15; 133(10): e170226. 10.1172/JCI170226
(https://doi.org/10.1172%2FJCI170226)

(2) Brain. 2011 Jul; 134(7): 1959-1971. 10.1093/brain/awr148
(https://doi.org/10.1093%2Fbrain%2Fawr148)

(3) Mol Ther. 2016 Feb;24(2):287-297. doi: 10.1038/mt.2015.187. Epub 2015 Oct
6.

 

For more information and interviews, please contact:

Priscilla Assumpção

priscilla@sg-branding.com (mailto:priscilla@sg-branding.com)

This information is provided by Reach, the non-regulatory press release distribution service of RNS, part of the London Stock Exchange. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact
rns@lseg.com (mailto:rns@lseg.com)
 or visit
www.rns.com (http://www.rns.com/)
.

RNS may use your IP address to confirm compliance with the terms and conditions, to analyse how you engage with the information contained in this communication, and to share such analysis on an anonymised basis with others as part of our commercial services. For further information about how RNS and the London Stock Exchange use the personal data you provide us, please see our
Privacy Policy (https://www.lseg.com/privacy-and-cookie-policy)
.   END  NRALPMRTMTITMJJ