GNW-Adhoc: ALSA Ventures founds new gene therapy portfolio company Axovia Therapeutics to treat ciliopathies

^* 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

[email protected]

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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