Coave Therapeutics

PDE6b-related Retinitis Pigmentosa is a rare inherited retinal dystrophy affecting up to 3,000 people in the US Symptoms often start in childhood leading to blindness by midlife with no approved therapy HORA-PDE6b has demonstrated clinically meaningful benefits in visual functions and good safety profile in PDE6b patients at 24-month follow-up from ongoing Phase I/II trial Rare Pediatric Disease Designation provides eligibility to receive a Priority Review Voucher upon future marketing authorization of HORA-PDE6B, possible in 2028 December 17, 2024 – eyeDNA Therapeutics (‘eyeDNA’), a clinical-stage gene therapy company focused on developing life-changing therapeutics for inherited retinal disorders, announces that it has been granted a Rare Pediatric Disease Designation (RPDD) by the US Food & Drug Administration (FDA) for HORA-PDE6b, its novel gene therapy for patients with inherited retinal dystrophy (IRD) caused by mutations of the PDE6b gene. PDE6b Retinitis Pigmentosa (PDE6b RP) is a rare inherited retinal dystrophy that is estimated to affect up to 3,000 people in the US and 4,000 people in Europe, accounting for 1 to 3% of all RP cases. The disease is caused by mutations of the PDE6b gene resulting in dysfunctional PDE6b protein, disrupting the conversion of light into electrical signals and leading to the progressive loss of photoreceptors and vision. Symptoms often start in childhood and lead to blindness by midlife. There are currently no approved treatments for PDE6b RP. HORA-PDE6b is an AAV5-based gene therapy designed to deliver a non-mutated copy of the functional human PDE6b gene into the subretinal space, where it rapidly induces transgene expression and synthesis of functional PDE6b proteins in photoreceptor cells. By effectively providing these cells with a functional protein, HORA-PDE6b may significantly delay or halt retinal degeneration in PDE6b-deficient patients. eyeDNA presented positive 24-month follow-up results1 from its Phase I/II study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, USA in May. eyeDNA is exploring the optimal paths to making HORA-PDE6b available to PDE6b RP patients in the US and in Europe via an accelerated approval process. “PDE6b retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness with currently no treatments available,” said Rodolphe Clerval, Chief Executive Officer. “This Rare Pediatric Disease Designation (RPDD) for HORA-PDE6b from the FDA recognizes the urgent need for effective treatments for this devastating disease where early treatment could slow or halt disease progression and have a profound impact on patients’ quality of life. We look forward to providing further data updates from our ongoing Phase I/II trial with HORA-PDE6b, and we are particularly excited to see initial results in younger patients where HORA-PDE6b could produce the greatest clinical benefit.” Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions primarily affecting children under 18, with fewer than 200,000 cases in the US. Companies awarded an RPDD receive a rare pediatric disease Priority Review Voucher (PRV) from FDA when the designated drug is approved for the associated indication in the pediatric population. The voucher allows the recipient company to obtain FDA priority review of either a New Drug Application (NDA) or Biologics License Application (BLA) for a different product and/or indication, reducing the review time and accelerating any granted approval and subsequent market entry by at least four months. The PRV may be used by the original recipient, or it can be sold to another company for the purchaser’s use, with PRVs recently achieving prices above $100 million. Reference JB Ducloyer, et al. 12-month Safety and Efficacy Evaluation of HORA-PDE6b, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation (2024) Abstract 2134, Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting (Seattle, WA, USA) eyeDNA Therapeutics, a wholly owned subsidiary of Coave Therapeutics, is a clinical-stage gene therapy company, focused on developing life-changing therapeutics for inherited retinal disorders. Our lead program HORA-PDE6b, an AAV5-based gene replacement therapy, is being evaluated in a Phase I/II trial for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene (PDE6b RP) (NCT03328130). Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases. 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 coavetx.com and follow us on LinkedIn. The content above comes from the network. if any infringement, please contact us to modify.

Coave’s proprietary ALIGATER platform enables specific retargeting of AAV vectors to human receptors via a one-step chemical conjugation of peptide-based ligands at the surface of the capsid, bypassing the need for prior capsid modification ALIGATER offers unprecedent control over AAV and receptor interaction affinity, enhancing vector specificity and efficacy for systemic applications Paris, France, October 22, 2024 – Coave Therapeutics (‘Coave’), a genetic medicine company developing novel delivery vectors for the treatment of eye and neurodegenerative diseases, today announced the presentation of new data in a Late-Breaking Abstract at the 2024 European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, held in Rome, Italy. The data highlight the potential of Coave’s proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform to enable the creation of peptide conjugated AAV (coAAV) vectors that precisely target human receptors. Current gene therapy vectors often require high doses, leading to safety concerns due to low specificity, as well as elevated and increased manufacturing costs. Recently, several techniques have been developed to enhance vector tropism, such as genetically inserting peptides on the AAV capsid to modify their interactions with known cell receptors. While this technique is effective, it is limited on manufacturability and modularity, with constraints such as the size and presentation of the peptide. Coave’s ALIGATER platform enables the direct conjugation of peptide ligands to the surface of AAV capsids, through a streamlined, one-step chemical process. This innovation allows for the fine-tuning of peptide density and presentation on the capsid surface, optimizing receptor targeting without the need for capsid modification or changes in the existing CMC processes. The ALIGATER platform simplifies the engineering of both wild-type and de-targeted AAVs, offering flexibility in targeting specific human receptors, such as integrins expressed at the surface of muscle cells, or the transferrin receptor 1 (TfR1) to cross the blood-brain barrier. Dr. Lolita Petit, Coave’s Chief Scientific Officer, commented: “Our ALIGATER platform represents a game changer in AAV vector engineering. For decades, the gene therapy field has sought ways to rationally engineer AAV vectors towards specific receptors, to ensure increased specificity, safety and transability. ALIGATER provides unmatched flexibility, while maintaining CMC processes intact. Our exciting new data showcase the platform’s potential to significantly improve the efficacy and safety of gene medicine therapies.” Poster presentation details are as follows: Title: The Advanced Ligand Conjugation (ALIGATER™) platform chemically redirects AAV and LNP vectors towards specific receptors via peptide-mediated targetingDate & Time: Wednesday October 23; 13.30-15.00 CEST Poster ID: #P0074Presenter: Dr. Julien Spatazza, Senior Director of Discovery & Preclinical Development at Coave Therapeutics The abstract is available to registered delegates at ESGCT Congress . *** 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 across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent 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, Mark Swallow coavetx@medistrava.com

Paris, France, September 24, 2024 – Coave Therapeutics (‘Coave’), a genetic medicine company pioneering therapies for eye and neurodegenerative diseases, will present the first data on its peptide-conjugated vector technology, a key new component of its ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform technology at upcoming conferences. This breakthrough technology not only enables the redirection of AAV (adeno-associated virus) and LNP (lipid nanoparticle) vectors to specific receptors of interest but also the modulation of vector/receptor affinity, offering enhanced gene delivery option for precision medicines. CEO Rodolphe Clerval will present the Company’s progress at the following investor conferences: Chardan’s 8th Annual Genetic Medicines Conference – September 30 – October 1 New York City, NY, USAPresenting on September 30 at 13.30 EDT Jefferies London Healthcare Conference – November 19-21 London, UKAvailable for investor meetings In addition, Coave will present new scientific data from its ALIGATER™ platform at the following scientific, medical and regulatory conference: European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress – October 22-25 Rome, ItalyPoster title: “The Advanced Ligand Conjugation (ALIGATER™) platform chemically redirects AAV and LNP vectors towards specific receptors via peptide-mediated targeting.”Presenting on 23 October, 13.30-15.00 CEST (Poster ID# P0074) *** 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