About us

At Coave Therapeutics, we are leading the transition of genetic medicine from (ultra) rare to prevalent conditions, with an initial focus on neurodegenerative diseases.
*Co·av·e
(noun) from Conjugated AAV Vectors
(pronounced ‘cove’)
Our proprietary ALIGATER™ (AAV-Ligand Conjugate) platform introduces chemical modifications to AAV capsids, overcoming the limitations of current vectors on efficacy, safety, and manufacturability. With low doses and optimized routes of administration, our conjugated AAV vectors have demonstrated markedly improved transduction and biodistribution in the central nervous system and the eye across different species.
Our diversified 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.
We are backed by leading international life science investors:

Our team

Coave Therapeutics has an experienced leadership team with years of successful company building and gene therapy experience, as well as strong relationships with gene therapy academic and research centers of excellence to execute on its strategy.
The Coave Leadership Team

RODOLPHE CLERVAL, MSc CEFA
Chief Executive Officer

Rodolphe has over 20 years’ experience in the pharma and biotech industry. Rodolphe joined Coave Therapeutics in 2020 from Enterome, which he co-founded. During his tenure at Enterome, Rodolphe executed over 15 transactions, including major industrial partnerships with Takeda, J&J, BMS, Abbvie and Nestle Health Sciences, generating over €100 million in upfront, R&D payments, and equity investments. He was also actively involved in multiple fundraising rounds. Rodolphe has previously worked in business development at TcLand Expression and Genzyme, and as a sell-side equity analyst at Natixis Bank.
Rodolphe graduated with a M.Sc. degree in Biochemical Engineering from Polytech Marseille and is a Certified European Financial Analyst from EFFAS-SFAF.

THOMAS BLAETTLER, MD
Chief Medical Officer

Thomas has over 25 years’ experience in neuroscience, both in clinical residency and within the industry. Thomas joined Coave from Orphazyme A/S where he served as Chief Medical Officer until November 2021. At Orphazyme A/S he was responsible for devising the clinical development strategy and progressing the company’s rare neurodegenerative pipeline, while also contributing to both IPOs on the Copenhagen and Nasdaq stock exchanges. Thomas has also held global leadership roles within the clinical neuroscience divisions at both Roche and Bristol Myers Squibb, with a further neuroscience translational medicine role at Novartis.
Thomas completed his Doctor of Medicine at the University of Zurich completing 10 years of clinical residency and research in the neurology field. Thomas gained board certification from the Swiss Society of Neurology in 2003.

PATRICIA FRANÇON, PhD
Chief Operating Officer

Patricia has over 20 years’ experience in the biotech industry leading R&D, preclinical and clinical operations as well as manufacturing for the accelerated development of innovative biologics, including advanced cell & gene therapies. Patricia joined Coave from Skinosive where she served as Chief Operating/Technology Officer until November 2021. At Skinosive she managed all operational aspects of the business, proactively driving the company towards achieving its development goals.
Patricia obtained her PhD in Molecular and Cellular Biology from Paris VI University and completed her postdoctoral research at McGill University.

GAELLE LEFEVRE, PhD
Head of Discovery Sciences

Gaelle joined Coave Therapeutics in 2019 as a project manager. She holds a Ph.D. in Molecular Oncology from Paris University and spent over 20 years as a research scientist in renowned academic institutions in France (Institut Pasteur), Canada (McGill University) and the US (NIH, University of Michigan). In 2014, she transitioned to project management and coordinated several large, collaborative translational research studies on rare genetic sensory disorders of the eye and ear and the development of AAV-mediated gene therapies for these indications. She brings her unique expertise to the global management of all the non-clinical projects at Coave Therapeutics.

CEDRIC COUSTEIX, MSc
Finance Director

Cédric brings 15 years of experience in financial auditing, management, and transaction consulting. Prior to joining Coave Therapeutics, he was at EY in Paris, where he specialized in private equity and growth companies and acquired deep expertise in financial auditing and transaction consulting. Cédric is an engineer who graduated from the Grande École d’Ingénieurs (Institut supérieur d’électronique de Paris – ISEP) in Paris and holds a degree in corporate finance from the EM Lyon Business School.

CATHERINE MATHIS, PharmD
Vice President Regulatory Affairs

Catherine joined Coave in September 2022 and brings over 30 years’ experience in development and regulatory affairs in the pharmaceutical and biotech industries. Catherine spent 20 years at Transgene as Senior Director, Head of Regulatory Affairs setting up and leading the regulatory affairs department and supervising global regulatory activities for the development of gene therapy products in genetic diseases and immuno-oncology. Catherine has also held senior leadership and regulatory roles at Voisin Consulting Life Sciences, TxCell (a subsidiary of Sangamo Therapeutics), Elsalys Biotech and most recently Enterome for the development of cell therapy products, monoclonal antibodies and peptide based vaccines, respectively, in oncology and GI diseases fields.

Catherine holds a PharmD and a Master’s degree in applied and basic toxicology from Paris University. She began her career in clinical research roles at Ipsen and Sanofi Pasteur.

JULIEN BERGER
Head of Legal Affairs

Julien has almost 20 years of global, regional and local healthcare legal affairs experience among corporate, commercial, medical, R&D, clinical operations and M&A activities. Julien joined Coave in May 2022 from Galapagos where he served as Senior Legal Counsel Director providing legal support and advice to its global teams.

Prior to Galapagos, Julien spent over 15 years in the legal team at Genzyme, most recently as Legal Director at Sanofi Genzyme following its acquisition. Julien developed and led the legal department for Genzyme’s French entity, supporting the growing business in five therapeutic areas, managing the launch of products in addition to supporting Genzyme’s acquisition by Sanofi.

Julien is a Business Law graduate from the universities of Lyon and Strasbourg.

The Coave Team

NICOLE BRUMENT, MSc
Head of CMC and Supply Chain

Nicole was a BioProcess Engineer at INSERM Translational gene therapy for genetic diseases -UMR 1089 for 18 years managing the development of production processes for viral vectors, especially for AAVs. She was involved in the early development of Coave Therapeutics’ lead products, for process design as well as for GMP manufacturing (2 processes patented). She also worked for a year in Florida at Biologix, US – now Thermo Fisher Scientific. Nicole began her career in the pharmaceutical industry at GSK in cardiovascular research. She obtained a master’s degree from the University of Paris.

EMMANUELLE JORDI, PhD
CMC Project Manager

Emmanuelle joined Coave Therapeutics in 2020 as a CMC project manager. She has 7 years’ experience in the non-clinical development of biologics for both European and American biotechnology companies, with a strong focus in CMC and regulatory affairs of gene therapy. In her previous company, Leads to Development (L2D), Emmanuelle has been the CMC project lead for 3 non-viral gene therapy products in oncology on behalf of L2D’s clients, which successfully reached clinical stage Phase I and II. She has developed a broad understanding of agency expectations and operational creativity to face development challenges of early-phase products.
Emmanuelle holds a Ph.D. in Neuroscience from The Rockefeller University, USA and The University Pierre et Marie Curie, France.

MARIE-ANNE BURLOT, PhD
Preclinical Manager

Marie-Anne joined Coave Therapeutics in January 2021 as a project manager. She has a veterinary medicine degree from the National Veterinary School of Toulouse and holds a Ph.D. degree in Neuroscience from Paris University. During her thesis, she used AAV vectors to restore brain cholesterol metabolism in a model of Alzheimer’s disease (AD). In 2014, she joined Animalliance to practice as a veterinarian in experimental research. In 2016, she transitioned to project management and coordinated several research studies on AD, brain aging and neurodegenerative diseases at Medday Pharmaceuticals.

CORNELIE BATISSE, PhD
Quality Assurance Manager

Cornélie Batisse joined Coave Tx in July 2021 as Quality Assurance manager. She holds an engineering degree from Chimie ParisTech and a PhD degree in therapeutic chemistry from Paris Descartes university. In 2015 she joined Biotech Quality Group to gain experience in the pharmaceutical industry, particularly in QA. Prior to joining Horama, she was fully involved in the development of a Quality Managment System at Medday Pharmaceuticals, supporting various processes such as R&D and Temporary Authorization for Use holder activities.

MAGALY LEVEQUE
Office manager

Magaly joined Coave Tx in June 2017. Previously she used to work in the furnishing world for almost 20 years as commercial assistant (Boussac, Pierre Frey) before becoming the office manager of the French subsidiary of Designers Guild. She holds a degree in business and administration.

Our Board of Directors

FREDERIC CHEREAU, MBA
Chairman

Frederic currently serves as the President and Chief Executive Officer of LogicBio (Nasdaq: LOGC), a clinical stage genetic medicines company pioneering gene editing and delivery platforms to address rare and serious disease. He has more than 25 years of executive leadership and industry experience in biotechnology, pharmaceuticals, and medical devices.
Prior to LogicBio, Frederic has worked at Genzyme, Pervasis Therapeutics, Shire and aTyr Pharma as General Manager, President & CEO, Senior Vice President and COO respectively. Frederic holds a BSc in Physics, an MSc in Management and completed his MBA at INSEAD, Fontainebleau, France.

GUILLAUME BLAVIER
Omnes Capital

Guillaume has been in the life sciences venture space since 2018, holding roles at Omnes Capital and Merieux Equity Partners. Prior to this, he held research and quality roles at Johnson and Johnson, Rouen University Hospital-Charles Nicolle (CHU de Rouen) and Sanofi.
Guillaume holds a Master’s degree in Business Development from Audencia Business School and holds a diploma in Pharmacy from Rouen University.

JEAN-FRÉDÉRIC CHIBRET
Théa Open Innovation

OLIVIER DANOS, PhD

Dr. Danos has over 30 years’ experience in gene therapy, as a scientist pioneering gene transfer technologies and as a leader in the biotech and pharmaceutical industry. He currently serves as the Chief Scientific Officer (CSO) of RegenxBio (Nasdaq: RGNX), a clinical stage biotechnology company developing a proprietary adeno-associated virus (AAV) gene delivery platform for the treatment of retinal, neurodegenerative and metabolic diseases.
Prior to RegenxBio, Olivier has undertaken a number of clinical research and executive leadership roles including Senior Vice President at Biogen and Kadmon Pharmaceuticals, Director of the Gene Therapy Consortium at UCL, CSO at Genethon, as well as Director of Research at the CNRS (Centre national de la recherche scientifique – The French National Centre for Scientific Research) and Principal Investigator at Institut Pasteur. Olivier completed his PhD at the University Paris Diderot.

CLAUDIA MITCHELL, PhD MBA

Claudia is a scientist and entrepreneur with over 20 years’ research and industry experience. She most recently served as Senior Vice President of Portfolio Strategy at Astellas Pharma in Tokyo, Japan. Prior to this, Claudia co-founded Universal Cells, where she served as CEO until it was acquired by Astellas for over $100 million. Claudia also co-founded Halo-Bio RNAi Therapeutics and served as its Chief Scientific Officer.
Claudia holds a PhD in Molecular Biology from the University of Paris and an MBA in International Management from Ecole des Ponts Business School, Paris, France. She was awarded the 2018 EY Entrepreneur of the Year award in Life Sciences for the Pacific Northwest Region, USA.

BRUNO MONTANARI, PharmD MSc
Seroba Life Sciences

Bruno is a Partner at Seroba Life Sciences. He has a background in venture capital and investment banking, with a focus on the pharmaceutical, biotechnology and medical device industries.
Bruno graduated in 1998 with a PharmD, from the Université René Descartes, Paris V and in the same year completed a master’s degree in Strategic Management at HEC.
Prior to joining the firm in 2017, Bruno was a Partner at Omnes Capital (Paris), in charge of life sciences investments for the venture capital team. His previous venture capital experience was at Atlas Venture (Paris/London) and CDP Capital (Paris/Montreal). He started his career in 1999 in London, in the healthcare teams of the investment banking divisions of Deutsche Bank and later Merrill Lynch. Bruno brings a wealth of experience and strong networks, particularly in continental Europe, where he is based.

PHILIPPE MONTEYNE, MD PhD
Fund+

Philippe is a Partner at Fund+, a Belgian venture capital. He has held senior positions in SmithKline Beecham, GSK, and Sanofi. He served as the Head of Global Vaccine Development at GSK for several years, and then served as Senior VP-Head of Development and CMO at GSK Rare Diseases from 2010 to 2012. More recently, he served as Vice President R&D France at SANOFI. He is a director of different Biotech companies. Philippe was also visiting Professor of Neurology at the UCL (Université Catholique de Louvain), Brussels.
He holds an M.D. degree in 1989 and Ph.D. in Viral Immunology in 1995, both from University College London, and has also been trained at Pasteur Institute in Paris.

WILLEM BROEKART, MSc PhD
V-Bio (Observer)

Willem is the Managing Director of V-Bio Ventures, a life sciences venture capital fund. Before co-founding V-Bio Ventures in 2015, Willem spent over 15 years working in and growing venture capital-backed life sciences companies as an entrepreneur, consultant, and executive. He was the founder and CEO of Fugeia NV (now owned by Cargill), and Vice President R&D of CropDesign NV (now part of BASF). Before joining CropDesign NV, Willem was a professor of plant biotechnology and plant pathology at the University of Leuven, Belgium.
Willem holds an M.Sc. and Ph.D. in Bioscience Engineering from the University of Leuven, Belgium, and a master’s degree in agricultural economics from IGIA/ESSEC in Paris, France.

THIERRY LAUGEL, PhD MBA
Kurma Partners (Observer)

Thierry is the managing partner of Kurma Life Science Partners. Thierry holds a Ph.D. in pharmacology and an MBA from INSEAD. Thierry was a scientific representative for Laboratoires Fournier in Japan for four years, at a time when Lipanthyl was released in Japan and research programs were being rolled out with Grelan, a Takeda Group company. He subsequently joined Flamel Technologies after its IPO on Nasdaq, as project director for Pharmaceutical R&D. In 1998, he moved to the investment world, firstly at CDC Innovation and then with AGF Private Equity, where he supervised the healthcare investment team. He subsequently co-founded Kurma Life Sciences in 2008. Thierry has led investments in more than 25 companies including Actelion, Arpida, Targacept, Adocia, Auris and Erytech.

RODOLPHE CLERVAL, MSc CEFA
Chief Executive Officer

Rodolphe has over 20 years’ experience in the pharma and biotech industry. Rodolphe joined Coave Therapeutics in 2020 from Enterome which he co-founded. During his tenure at Enterome, Rodolphe executed over 15 transactions, including major industrial partnerships with Takeda, J&J, BMS, Abbvie and Nestle Health Sciences, generating over €100 million in upfront, R&D payments, and equity investments. He was also actively involved in multiple fundraising rounds. Rodolphe has previously worked in business development at TcLand Expression and Genzyme, and as a sell-side equity analyst at Natixis Bank.
Rodolphe graduated with a degree in Biochemical Engineering from Polytech Marseille and is a Certified European Financial Analyst from EFFAS-SFAF.

Our Scientific Advisory Board

Our Scientific Advisory Board (SAB) is a group of world-leading experts in neurodegenerative diseases, neurosurgery, autophagy and gene therapy.

ANDREA BALLABIO, MD
Director of Telethon Institute of Genetics and Medicine (TIGEM), Italy

ERWAN BÉZARD, PhD
Research Director, Institute of Neurodegenerative Diseases, University of Bordeaux, France

OLIVIER DANOS, PhD
Chief Scientific Officer, RegenxBio

THOMAS GASSER
Head of Neurology, University of Tuebingen, Germany

STEPHANE PALFI
Head of the Neurosurgery Department at Henri Mondor Medical justify, Paris University (UPEC), France

MARK RICHARDSON, MD PhD
Charles A. Pappas Associate Professor of Neurosciences, Harvard Medical School, US

ANTHONY SCHAPIRA
Head of Department of Clinical Neuroscience at UCL Institute of Neurology (ION)

LAMYA SHIHABUDDIN, PhD
Chief Scientific Officer, 4:59 NewCo, a 5AM Ventures Company

DAVID STANDAERT, MD PhD
John N. Whitaker Professor & Chair of Neurology Clinic, UAB Hospital, Birmingham, Alabama, US

Our Collaborations

At Coave, we welcome the opportunity to partner with leaders in gene therapy, leveraging our proprietary next-generation ALIGATER™ (AAV-Ligand Conjugate) platform to enable targeted delivery and enhanced gene transduction to improve the effectiveness of advanced gene therapies for neurodegenerative and ocular diseases.

ABL is a pure play contract development and manufacturing organization (CDMO) specialized in the development and manufacturing of viral vectors for vaccine candidates, gene and cancer therapies.
We are collaborating with ABL to develop gene therapy manufacturing processes that enable us to control the development and manufacture of our pipeline products in an end-to-end manner.
Initially we will co-develop manufacturing technologies for AAV-based gene therapy products, after which we have an exclusive option to secure process development capacity and laboratory space within ABL’s facility. This will enable us to further develop and scale-up manufacturing technologies for our next generation AAV vectors and AAV-based products, including its ALIGATER platform.
ABL and Coave’s process development teams are working jointly in ABL’s state-of-the-art GMP facility in Lyon (France).
For more information see the press release announcing the collaboration here.

The Institute of Neurodegenerative Diseases (IMN) is a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS). The IMN, established in January 2011, was founded by Dr Erwan Bézard to develop new therapeutic approaches for neurodegenerative diseases by facilitating translational research from the laboratory to the patient bed.
We are collaborating with IMN to identify new targets and pathways associated with neurodegeneration and leverage our expertise in gene therapy, including our AAV-Ligand Conjugate (ALIGATER) platform to develop coAAV based gene therapy products for neurodegenerative diseases. Our collaboration with IMN will initially focus on developing coAAV based gene therapy products for the targeted delivery of TFEB to deep brain structures for the treatment of various neurodegenerative disorders, focusing initially on Multiple System Atrophy (MSA) and idiopathic Parkinson’s disease (PD) (a-synucleinopathies).
IMN will be responsible for carrying out in vivo studies to validate the concept of gene therapy for TFEB delivery in models of MSA and PD. The collaboration aims to generate further in vivo proof of concept data and enable the selection of therapeutic candidates to enter Investigational New Drug (IND) enabling studies.
For more information see the press release announcing the collaboration here.

Technology

Our ALIGATER™ (AAV-Ligand Conjugates) platform leverages the wealth of knowledge around commonly used and well-characterized AAV vectors with a proprietary chemical conjugation technology to produce novel coAAV capsids for gene therapy products.
AAV-Ligand conjugation technology is altering the biology of AAV capsids through several potential mechanisms:
BIODISTRIBUTION

Site-specific conjugation onto capsid proteins enables altering of extracellular capsid sequestration, by blocking the binding of the AAV capsid to extracellular motifs.

CELL AND TISSUE TARGETING

Ligands are rationally designed to improve cell and tissue targeting, based on defined ligand-receptor interactions.

INTRACELLULAR

Site-specific conjugation of capsid proteins leads to improvement of intracellular AAV capsid trafficking and payload delivery to the nucleus.

EVADE IMMUNE RESPONSES

Ligand conjugation reduces coAAV exposure to immune reaction and neutralizing antibodies.

Preclinical experiments have demonstrated superiority of coAAVs over best-in-class AAV serotypes in terms of transduction efficiency and tissue distribution.
As a result, our ALIGATER technology offers the potential to generate coAAV based gene therapies with improved delivery to target tissue, at lower doses, thus limiting systemic and local toxicity.

Manufacturing

A key element of our strategy is to develop a robust manufacturing process for our next-generation AAV-based gene therapies.
We have a growing team of in-house experts who have made significant advances in process and product development for our proprietary next-generation ALIGATER™ (AAV-Ligand Conjugate) platform. Core elements of these processes are protected by patents and proprietary know-how.
We are collaborating with ABL, a CDMO with proven expertise in developing and executing GMP manufacturing of viral vectors for gene therapy products, to co-develop manufacturing technologies for AAV-based products, including those deriving from our proprietary next-generation ALIGATER platform.
Coave and ABL’s process development teams are working together in ABL’s state-of-the-art GMP facility in Lyon (France), which will give us the flexibility and capacity to manufacture high-quality gene therapy products at large scale.

Pipeline

We are developing a diversified pipeline of novel genetic medicines for neurodegenerative diseases and ocular diseases, by harnessing our ALIGATER™ technology to enable precise and safe delivery of low dose gene therapy. We are targeting genetically and non-genetically defined diseases by correcting, activating or regulating key functions and pathways involved in the pathogenesis of these diseases.

Neurodegenerative Diseases

Targeting Protein Degradation by Activating the Autophagy Lysosomal Pathway

The misfolding and accumulation of disease-related proteins are common hallmarks of several neurodegenerative diseases. As examples, beta-amyloid and tau in Alzheimer’s disease, alpha-synuclein (aSyn) in Parkinson disease and related disorders, and mutated huntingtin in Huntington’s disease, are shown to contribute to disease progression and neurodegeneration.

Pathological accumulation of aSyn is the common distinguishing trait amongst the group of brain disorders known as synucleinopathies, which include Parkinson’s disease (PD), Dementia with Lewy bodies (DLB), and Multiple System Atrophy (MSA). These disorders progressively develop neuronal and glial inclusions enriched with misfolded, phosphorylated and insoluble aSyn.

Over the past decade, several treatment strategies directly targeting aSyn have been evaluated in preclinical and clinical studies. These diverse approaches include removal of aggregated aSyn with passive or active immunization or by expression of vectorized antibodies, modulating kinetics of misfolding with small molecule anti-aggregants, lowering aSyn gene expression by antisense oligonucleotides or inhibitory RNA.

More recently, several new approaches aiming to harness the natural cellular machinery for degradation of toxic protein aggregates, including aSyn, have been investigated.

The autophagy lysosomal pathway (ALP) is a central cellular pathway enabling the degradation of toxic protein aggregates. Key factors of the ALP, such as Glucocerebrosidase (GBA1) and Transcription Factor EB (TFEB), have been shown to play important roles in aSyn clearance mechanisms (Figure X).

At Coave, using our ALIGATER™ platform, we have generated coAAV based gene therapy delivering a safe, low dose of GBA1 and TFEB precisely in the targeted structures of the central nervous system (CNS), aiming to activate or restore the macro-autophagy and lysosomal functions to eliminate or prevent the accumulation of toxic protein aggregates associated with neurodegenerative disease. 

Parkinson’s Disease

Parkinson’s disease (PD) is a severe and progressive neurodegenerative disorder that affects more than seven million people worldwide.

Population-based genetic studies have recently identified several causative and risk genes for PD. Many of these genes are involved in the normal functioning of lysosomes, a cell organelle containing enzymes responsible for degrading biomolecules.

The GBA1 gene encodes the lysosomal enzyme beta-glucocerebrosidase (GCase), which is needed for the disposal and recycling of glycolipids — a type of cellular lipid component that is known to accumulate with aging. Mutations in the GBA1 gene lead to a deficiency of GCase and are associated with earlier onset of PD, with more severe symptoms, and increased likelihood of progression to dementia. Certain mutations in GBA1 reduces the functionality of GCase, which may favor toxic build-up of alpha-synuclein fibrils resulting from the accumulation of glycolipids.

There are currently no approved therapies that modify the course of PD or the underlying pathological process.

CTx-GBA1: an optimized gene therapy for treating GBA1 associated diseases

Our gene therapy candidate, CTx-GBA1, utilizes a coAAV vector to deliver a gene sequence encoding functional GCase enzyme. CTx-GBA1 has been optimized for improved transduction and distribution of the GBA1 gene in the key structures (basal ganglia) of the brain involved in Parkinson’s disease and related disorders.

Multiple System Atrophy and other a-synucleinopathies

The a-synucleinopathies, Multiple System Atrophy (MSA), Parkinson’s disease (PD) and Lewy Body Dementia (LBD), are characterized by aggregates of a-synuclein, associated with impairment of the autophagy-lysosomal pathway. TFEB is a master regulator of the autophagy lysosomal pathway, a central cellular pathway controlling the degradation of toxic protein aggregates. Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1.

Coave, in collaboration with the Institute of Neurodegenerative Diseases (IMN), is using its ALIGATER platform to generate coAAV based gene therapy products to target the delivery of TFEB in deep brain structures to treat various neurodegenerative disorders, starting first with a-synucleinopathies.

A paper2, authored by eminent scientists at IMN, including Erwan Bézard, IMN and INSERM Research Director and world-renowned Dr Andrea Ballabio, Scientific Director, Telethon Institute of Genetics and Medicine (TIGEM), demonstrated effective delivery of TFEB AAV-based gene therapy in disease models of MSA and PD.

CTx-TFEB: optimized gene therapy to treat a-synucleinopathies

Our gene therapy product candidate, CTx-TFEB, utilizes a coAAV vector to deliver a gene sequence encoding functional TFEB transcription factor. CTx-TFEB has been optimized for improved transduction and distribution of the TFEB gene in the key structure of the brain involved in MSA and PD.


References

  1. Martini-Stoica et al. The Autophagy-Lysosomal Pathway in Neurodegeneration: A TFEB Perspective. Trends Neurosci. 2016 Apr;39(4):221-234. https://doi.org/10.1016%2Fj.tins.2016.02.002
  2. Arotcarena et al. Transcription factor EB overexpression prevents neurodegeneration in experimental synucleinopathies, JCI Insight 2019 https://doi.org/10.1172/jci.insight.129719

Ocular diseases

Inherited Retinal Dystrophies

Inherited retinal dystrophies are rare ophthalmic pathologies that can be divided into two groups:

  1. Pigmentary retinopathies, which include retinitis pigmentosa (RP) and Leber congenital amaurosis
  2. Macular dystrophies

Retinitis pigmentosa is the most common form of inherited retinal dystrophy representing 50% of all retinal dystrophies.

While multiple genes are implicated in each of these groups, within each patient or family, only one causative gene is involved.

PDE6b Inherited Retinal Dystrophy (IRD)

PDE6b RP is an inherited retinal dystrophy that leads to blindness by midlife and is characterized by the progressive loss of photoreceptors, with or without the loss of retinal pigment epithelium cells.

It is caused by mutation of the PDE6b gene resulting in dysfunctional Rod PDE6, an enzyme found in rod outer segments that plays a key role in the phototransduction cascade in rods (the process by which light is converted into electrical signals). Dysfunction of the PDE6 protein, and in particular its PDE6ß subunit, ultimately leads to death of rod photoreceptor cells, then cone photoreceptor cells, leading to blindness.

Mutation of PDE6b is one of the most prevalent human mutations within autosomal recessive RP and accounts for 2-4% of RP cases.

There are currently no approved treatments for PDE6b RP.

CTx-PDE6b: A clinical stage AAV based gene therapy for treating PDE6b IRD

CTx-PDE6b is an AAV5 based gene therapy designed to deliver a full-length non-mutated copy of the functional human PDE6b gene into the subretinal space, where it rapidly induces robust transgene expression and synthesis of functional PDE6b proteins in photoreceptive rods and cones. By effectively providing these cells with a functional protein, CTx-PDE6b may significantly delay or halt retinal degeneration in PDE6ß-deficient patients.

CTx-PDE6b is currently in Phase I/II clinical trials.

A description of the protocol can be found on clinicaltrials.gov

Press Releases

October, 20 – 2022

September, 26 – 2022

September, 20 – 2022

September, 14 – 2022

September, 6 – 2022

June, 30 – 2022

June, 9 – 2022

May, 4 – 2022

April, 27 – 2022

April, 13 – 2022

November, 15 – 2021

October, 12 – 2021

September, 23 – 2021

September, 16 – 2021

July, 21 – 2021

Careers

JOB OPENINGS

We are a talented, passionate group of colleagues with a desire to translate innovative science into novel gene therapies for patients with neurodegenerative and ocular diseases and beyond.

We are committed to building a vibrant team combining deep expertise in AAV vector engineering and genetic construct design, innovative and advanced therapeutic product development, and manufacturing.

We are looking for more talented individuals to join our team.

Contact us

We welcome enquiries regarding our pipeline programs, ALIGATER technology, partnerships, and career openings.

Headquarters
63bis avenue Ledru Rolin
75012 Paris – France

Labs

INSTITUT DU CERVEAU ET DE LA MOELLE EPINIÈRE – ICM
Hôpital Pitié-Salpêtrière
47 bd de l’Hôpital
75013 Paris 

Business Development Enquiries
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