Coave Therapeutics Showcases its ALIGATER™ Platform for Generating Conjugated AAV (coAAV) Vectors Exhibiting Superior Performance in Delivering Ocular Gene Therapy through the Suprachoroidal Route
April 29 2024 - 6:00AM
coAAV-mediated gene transfer holds
promise for expanding the scope of ocular diseases treatable via
suprachoroidal administration
Data to be presented in oral session at
ASGCT 2024
Paris, France, April
29, 2024 – Coave Therapeutics
(‘Coave’), a genetic medicine company focused on developing
life-changing therapies, announces today that preclinical studies
on its novel Conjugated AAV (coAAV) gene therapy vectors
demonstrate promising results for ocular gene therapy. Engineered
coAAV vectors, generated using Coave’s ALIGATER™ platform, show the
ability to increase both the distribution and expression patterns
of transgenes delivered via suprachoroidal administration,
effectively improving the targeting of key tissues at the back of
the eye. This advancement suggests a new, potentially impactful
approach for treating acquired retinal diseases. These data,
generated in collaboration with REGENXBIO, will be presented at the
2024 American Society of Gene & Cell Therapy (ASGCT) meeting,
which will be held virtually and in person in Baltimore, MD, US May
7-11.
Oral presentation details are as
follows:
- Abstract Title:
Identification of Novel Ligand-Conjugated AAV Vectors with Enhanced
Properties for Suprachoroidal Gene Delivery
- Abstract Number:
86
- Date & Time:
Wednesday May 8, 2024; 1:30 p.m. – 3:15 p.m. ET
- Poster Session:
Ophthalmic & Auditory: Delivery Innovations
- Room: 318 -
323
- Presenter: Gaelle
Lefevre, VP Alliance & Portfolio Strategy at Coave
Therapeutics
Using its ALIGATER™ platform, Coave has
developed a range of new coAAV vectors, each specifically
engineered with a distinct set of small molecule ligands at its
surface through bioconjugation. These coAAVs have been screened for
administration to the suprachoroidal space (SCS) of the eye, an
attractive alternative to invasive subretinal administration to
deliver AAV-based gene therapies to the back of the eye. A library
of coAAV candidates was injected into the SCS of non-human primates
(NHPs) and evaluated in comparison to an unconjugated, benchmark
AAV capsid. Three weeks post injection, the distribution of vector
genome copies and the levels of transgene-specific expression were
analysed in ocular and peripheral tissues.
Several of Coave’s coAAVs showed more widespread
and enhanced expression in key target tissues of the eye, notably
the retina, versus the control vector, and lower off-target
systemic distribution to tissues such as the liver and kidney.
Coave’s coAAVs represent some of the first
engineered AAV vectors optimised for SCS delivery. These vectors
open new avenues for next-generation gene therapy vectors for
ocular administration, potentially eliminating the need for
invasive subretinal injections. The superior properties of coAAVs
for ocular gene transfer via SCS administration have the potential
to enable the development of more efficient, targeted, and
well-tolerated gene therapies for the treatment of common retinal
disorders.
Dr Lolita Petit, Coave’s Chief
Scientific Officer, commented: “Delivering gene therapies
to the posterior segment of the eye, notably the retina, poses a
key challenge: achieving broader distribution through a less
invasive procedure compared to the subretinal injection route,
while still ensuring sustained transgene expression in target
cells. This is an exciting time as our new data emphasize, for the
first time, the potential of improving the delivery of gene therapy
through the suprachoroidal space using Coave’s chemically
engineered coAAVs. This holds the potential to enable broader
applications in terms of scope of ocular diseases.”
Coave’s abstract (number 86) can be viewed on
ASGCT’s website (https://annualmeeting.asgct.org/).
***
About Coave
Therapeutics At Coave Therapeutics, we are leading
the transition of genetic medicine from rare to prevalent
conditions, starting with neurodegenerative and eye diseases. Our
proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform
introduces chemical modifications onto AAV capsids or Lipid
Nanoparticles (LNPs) to overcome the limitations of current vectors
on efficacy, safety, and manufacturability.
With low doses and optimized routes of
administration, our conjugated vectors have demonstrated markedly
improved transduction and biodistribution in the central nervous
system and the eye across different species. Our diverse pipeline
of novel genetic medicines can potentially transform the lives of
people afflicted by rare and prevalent neurodegenerative and ocular
diseases – including genetically and non-genetically defined
indications.
Headquartered in Paris, France, Coave
Therapeutics is backed by leading international life sciences
investors. For more information about the science, pipeline, and
people, please visit https://coavetx.com/ and follow us on
LinkedIn.
CONTACTS Coave
Therapeutics Rodolphe Clerval,
CEO contact@coavetx.com
MEDiSTRAVA Sylvie Berrebi,
Leila Adlam, Mark
Swallow coavetx@medistrava.com Tel: +44 (0)203 928
6900