Building companies
that deliver meaningful
results.
We are dedicated to provide high clinical benefits and financial returns, building innovative companies from the best research teams worldwide. We gather execution-focused teams to deliver meaningful results.
Opportunity in rare disease is real and strong.
The economics of rare diseases are fundamentally different. Higher pricing, higher success rates, and higher returns make it a uniquely attractive space for patients and investors alike.
218k€
Annual price per patient
Average annual price per patient with rare diseases vs 13k€ for common diseases.
2×
Likelihood of success
From Phase 1 to marketing authorization: 17% for orphan drugs vs 8% in general.
7.2×
Investment multiple
Average investment multiple from Phase 1 to approval in orphan drugs vs. 2.1× for non-orphan.
Our investment thesis
A disciplined, four-step approach to build impactful biotechs in rare diseases, from sourcing the best science to delivering real outcomes for patients and investors. We identify the best research teams and breakthrough innovation worldwide, with a sharp focus on unmet needs in rare diseases.
We assemble execution-focused teams, operators, scientists and investors, to bring programs to life. We advance programs with clinical rigor, leveraging our network of 6,000+ medical and scientific experts. We deliver high clinical benefits to patients and meaningful financial returns to investors.
A proven
track-record.
A relentless partner to address rare diseases.
Fighting rare diseases for 65+ years
A proven model
Meet our team.
A team of operators, scientists and investors with decades of combined experience in rare diseases.
Alexandre brings three decades of experience across pharma, biotech and investment banking. He is Head of Strategic Projects at Genethon and Managing Partner at Finpact.
He was Head of Business Development and Partnerships at Genethon. Before Genethon, Alexandre spent 20 years at Sanofi including ten years in finance across Europe and Asia and ten years in general management. He led the group in 25 acquisitions including Genzyme in 2011 and was CFO Asia.
Graduated from Institut Supérieur de Gestion (ISG) business school.
Pierre-Albert joined Ampleia at inception in 2023, bringing a decade of experience in biotech and finance.
Before Ampleia, Pierre-Albert was Business Development Director at Genethon, contributing to deals with Sarepta, Spark (acq. by Roche) and AveXis (acq. by Novartis). He was instrumental in the creation of Atamyo Therapeutics. He also negotiated deals for I-Stem, including a partnership with Urgo Group.
Started his career as an analyst at Clipperton Finance. Graduated from ESPCI Paris and ESSEC Business School.
Emilienne is Head of Development at Ampleia since July 2023. She joined from OSE Immunotherapeutics, where she was Director of Pharmaceutical Programs and Strategic Partnerships.
Prior to OSE, she was Director of Global Program Coordination at Nanobiotix. Before that, she held development roles at Gensight (gene therapy) and Sotio (cell therapy). She started her career at BioAlliance Pharma.
Pharm.D and Ph.D in Pharmaceutical Sciences from Paris Saclay University.
Marion is Head of Sourcing & Startups and joined Ampleia in 2023.
She brings 15 years of experience in lifescience, genetic and rare diseases and business development.
Prior to joining Ampleia, Marion was business development director at Imagine Institute. She led negotiations and contributed to the execution of 40+ partnerships with pharma companies and biotechs within the rare diseases field. She was in charge of Imagine’s Accelerator program, executing due diligence processes to bring projects from research stage to pre-seed funding.
In 3 years, Marion has supported 6 projects, which led to 2 start up creations. Marion started her career at Alcimed, a consulting firm dedicated to innovative industries. She was part of the healthcare team and supported healththech clients in their product development, market access plan and global development strategy. Marion was awarded her PhD in Genetics and Neurosciences from the University Pierre et Marie Curie. Her research on the genetics of Autism Spectrum Disorders, led to the identification of a new genetic syndrome (Intellectual developmental disorder, X-linked syndromic, Pilorge type) and to publications in top-tier journals. Marion also graduated from University Paris Dauphine/Mines Paris with a MSc in Innovation and Technology Management.
Laurent is the CEO of Accure Therapeutics, a Spanish biotech company developing small molecules for CNS diseases, including optic neuritis.
He co-founded Accure in 2018 and has since guided the company’s translational development, from the end of the research phase through to Phase II clinical trials. In 2022, Accure negotiated a licensing agreement for its lead optic neuritis program with Oculis, which led in January 2025 to the publication of positive results demonstrating the neuroprotective effect of the drug candidate in this indication and paving the way for its potential use in future neurodegenerative disorders.
Laurent previously served as CEO of Sensorion Pharmaceuticals from 2012 to 2017.
He began his career at Merck KGaA and Roussel-Hoechst, where he held various positions in marketing and product strategy between 1985 and 2000, before joining Roche as part of the Corporate Business Development team.
In 2008, he joined Pierre Fabre as Head of Corporate Licensing & M&A. Laurent is a medical doctor specialized in public health.
Jean-Francois Briand is Operations and Innovation Director at AFM-Telethon.
He joined the association in 2011 and held several leading positions in the identification and selection of world-class projects in rare diseases. His team is financing and managing the exploitation of 350 research projects across international academic institutions and biotechs with the ambition to further push boundaries in disease understanding and new technologies emergence for rare disorders.
Jean-Francois started his career as scientist at the Center for Atomic Energy before joining biotech industry where he was managing molecular biology departments and developing various research projects in recombinant protein production, infectious diseases and oncology programs development.
Jean-Francois holds a PhD in microbiology and virology from Sorbonne University.
Ralph brings to Ampleia his long and successful experience in pushing drugs from discovery to commercialization.
Ralph is CSO at Neuvasq, a privately held research company focusing on the discovery and development of therapies to safeguard or rebuild CNS barriers. Prior to that, Ralph was CSO at Lysogene, a clinical-stage listed company developing gene therapies for lysosomal storage disorders. Before joining Lysogene, Ralph was Senior Vice President at Teva, in charge of small molecule Discovery and Product Development. Through his leadership, his team contributed to FDA approval of three drugs for neurological diseases and the clinical development of several programs covering a wide range of therapeutic areas, incl. neurological, inflammatory, respiratory and oncological diseases. He joined Teva from IRBM, where he was the Scientific Director. Prior to that, he was Head of Pharmacology at IRBM-Merck Research Laboratories Rome. Over his career, Ralph contributed to the discovery and/or development of several commercialized products, including Austedo® (for tardive dyskinesia and Huntington’s disease chorea) and Isentress® (for HIV-AIDS).
Ralph is board member at EG427 and scientific advisor of several biotechs, disease foundations, and academic institutions. He holds a Ph.D in biochemistry from The Hebrew University of Jerusalem.
Serge Braun is Scientific director at AFM-Telethon and President of Genosafe.
Prior to joining AFM-Telethon, Serge was Vice-President of Research at Transgene, a biotech dedicated to gene therapy and immunotherapy for cancer and infectious diseases. He started his career in academic research institutions both in France and the United States, focusing his works on neuromuscular diseases.
Serge co-founded Neurofit, a CRO specialized in preclinical tests on the central and peripheral nervous system, and was Vice-President of the Alsace BioValley bioparc, a major biotech hub in Europe, for several years. Serge is member of the National Academy of Pharmacy and scientific board members in various institutions, biotechs and VC firms. Serge holds a Pharm.D and Ph.D from Louis Pasteur University on neuromuscular diseases and immunotherapies.
Active programs.
Our portfolio companies are currently in stealth mode — building the next generation of therapies for rare diseases.
First-in-class therapeutic targeting novel metabolite transport.
Status
Active
Gene-therapy platform for large payload delivery.
Status
Active
Latest news.
Insights, milestones and announcements from Ampleia and our portfolio companies.
Let’s work together.
Tell us more about your project, needs and how Ampleia can support you to treat rare diseases.
