^* Development of the first gene therapy to treat diseases caused by
a dysfunction of the cilia can be caused
* AXV101 targets retinal dystrophy in patients with
Bardet-Biedl syndrome (BBS).
* FDA orphan and orphan drug designation
pediatric diseases
*First human study planned to begin in late 2024/early 2025
* Six potentially transformative therapies for ciliopathies are in the pipeline
pipeline
LONDON, Sept. 20, 2023 (GLOBE NEWSWIRE) — ALSA Ventures
(https://www.alsaventures.com/), a UK resident
Therapeutics-focused venture capital fund announces the takeover of Axovia
Therapeutics Inc. and the creation of a new portfolio company, Axovia
Therapeutics Ltd (https://axoviatherapeutics.com/).
Axovia is developing the first gene therapies for ciliopathies and has a
pipeline of products for these devastating diseases, including Bardet
Biedl syndrome (BBS).
The ALSA Ventures investment team has a plan to accelerate
Development designed to be the main program AXV101 in the next 18-24 months
into clinical trials and a quick route to clinical testing
Find proof of concept and approval.
AXV101 is an AAV9-based gene therapy for the treatment of BBS
associated retinal dystrophy in patients who are biallelic
Carrying mutations in the BBS1 gene. It is said to stop retinal degeneration
begins in childhood and leads to blindness by the age of 20.
Revised epidemiological analyzes suggest that 1 in 70,000 to
1 in 100,000 people in Europe and North America have BBS, and it
There is no treatment for retinal degeneration.
Alek Safarian, CEO of ALSA Ventures, said they are pleased to bring AXV101 into the
to bring clinical trials where there is access to well-characterized and
motivated patients will have.?Especially pediatric ones
Patients need early treatment before permanent vision damage occurs
occur,” he commented.
“Our investment team has identified Axovia Therapeutics as a valuable addition to our
growing company portfolio selected because of the preclinical results
are impressive, Axovia’s investigational drug has rare status
pediatric disease and has a proven AAV
transport mechanism,” he said.
The company is well positioned to provide a Priority Review Voucher (PRV).
FDA, which is awarded to sponsors, the drugs for
diseases, including rare pediatric diseases.
Axovia is based on decades of work by co-founders Professor Phil
Beales and Dr. Victor Hernandez at University College London in the field of
Ciliopathies.
Acting CEO of Axovia, Professor Phil Beales, said that the
Axovia’s gene therapy platform brings hope to BBS patients worldwide.
“In preclinical studies, our novel BBS1 gene therapy has
Basic disease of BBS changes and the loss of vision by stopping the
retinal degeneration,” explained Professor Beales.
Professor Beales is a renowned scientist and leading diagnostician
in the field of ciliopathies. He graduated from University College London (UCL)
Institute of Child Health provides research and patient care for one of the
the world’s most debilitating ciliopathies – BBS.
Find community resources
here: https://axoviatherapeutics.com/get-support-for-bbs/
“Our novel gene therapy uses an adeno-associated virus (AAV9) to…
“to introduce a functional copy of the faulty BBS gene into important tissues,”
he said.
?Since AAV is not known to cause disease in humans and strictly
can be controlled (it does not multiply like disease-carrying ones
viruses), it is the method of choice for gene transfer for numerous
Therapies, including Luxturna for retinal diseases.”
The available clinical data from more than 3,000 people who have a
over a period of more than 20 years show that AAV
Gene therapy is well tolerated and effective.
We thank Fieldfisher LLP for their assistance with this transaction.
For media inquiries please contact:
David James
Digital Mantra Group
About Axovia Therapeutics
Axovia Therapeutics, a portfolio company of ALSA Ventures, is developing the
first gene therapies for ciliopathies and has a pipeline of
products for these devastating diseases, including Bardet-Biedl syndrome
(BBS).
Ciliopathies are a group of more than 40 rare genetic diseases
Hereditary diseases that are linked to more than 950 genes that affect the function of the
affect cilia. These microscopic, finger-like organelles protrude
from most cells in the body.
The lead program, AXV101, is expected to enter clinical trials in the next 18-24 months
go to the exam.
AXV101 is an AAV9-based gene therapy for the treatment of BBS
associated retinal dystrophy in patients who are biallelic
Carrying mutations in the BBS1 gene. It is said to cause the death of the photoreceptor cells and
stop the degeneration of the retina.
BBS is a rare disease affecting between 1 in Europe and North America
70,000 and 1 in 100,000 people are affected. Currently there is none
Remedy.
For more information, visit https://axoviatherapeutics.com/.
About ALSA Ventures
ALSA Ventures is an early-stage life sciences investor. The
Company builds a business portfolio based on innovative
Findings are based on the treatment of diseases for which it is currently only available
There are limited or no therapeutic options. We work with one
broad global network of preclinical and clinical experts
a deep understanding of the disease biology relevant to implementation
and then the collective knowledge and operational experience of the team
to identify opportunities for drug interventions.
Our focus is to improve patient outcomes by
we exploit the full potential of active ingredients in the clinical stage.
As we continue to expand our portfolio, our mission remains clear: Us
want to bring life-changing therapies to patients worldwide.
For more information, visit https://www.alsaventures.com/.
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