Ampleia

Last week, Ampleia's team had the chance to regroup in the beautiful Ville de Pornic on the seafront for its annual seminar. It was a great occasion to look back on progress made since the inception of Ampleia and share perspectives on financing environment, trends in pharmaceutical discovery & development for rare diseases and deal-flow priorities for the upcoming year. We all jump on the opportunity of this beautiful location and wonderful weather to enjoy some times together and build-up relationships. #rarediseases #seaside #seafood #vc #lifescience # Alexandre Lemoalle, Marion Pilorge, Ph.D, Emilienne Soma, Ulku Cuhadar, PhD, MBA

Today, we are delighted to announce that Ralph Laufer joins Ampleia as Advisor and permanent member of its Investment Committee. "I am delighted to support Ampleia’s team as they deploy their ambitious mission to translate more treatments to the clinic for rare diseases patients. After 35+ years in the drug development space and spanning from discovery to approval, it is my hope to offer some of this expertise to select the most promising drug candidates and support exceptional scientists and entrepreneurs on their way to bring innovative and meaningful therapeutics to patients in desperate need." Ralph said. "We are grateful to welcome Ralph and benefit from his life-long and successful experience in pushing drugs from discovery to commercialization." Pierre-Albert mentioned. "Pharmaceutical development is a complex journey that leaves no room for improvisation. Seasoned developers who have gone through the whole development like Ralph are very scarce and we feel humble to collaborate with him on our companies." Ampleia is a venture-builder dedicated to rare diseases. Founded by AFM-Téléthon in 2023, it is dedicated to identify world-class research projects with strong translational potential and transform them into therapeutic options through company creation and venture financing. Ampleia is technology- and indication-agnostic and benefits from the network and the expertise of its founder to scout, assess and develop a portfolio of rare diseases companies. #rarediseases #venturebuilder #biotech #lifescience

Coming back from Bio€quity full of energy and enthusiasm! Marion Pilorge, Ph.D, our Head of Sourcing & Startup, was in San Sebastian earlier this week to meet with entrepreneurs, scientists and VCs in lifesciences. It was great to reconnect with former colleagues, friends and partners and meet new faces for future projects! #bioequityeurope #lifescience #rarediseases

We are delighted to see the research works from our analyst Ulku Cuhadar, PhD, MBA awarded by a publication in Cell Reports on the molecular behaviors of trans-synaptic proteins and how one of these, LGI1, can be critical for the synaptic function. Congrats Ulku! https://lnkd.in/eP4ZXqqf

Glad to have our COO Pierre-Albert Colcomb sharing his thoughts on academia/industries partnerships and challenges these alliances represent to get research turned into therapeutic options for patients. Thanks to LES France for the invite!

RARE DISEASE DAY 2023 was another great year for rare disease innovation. Of the 55 products that the FDA approved in the year, more than half of these were for rare diseases. We are pleased to see new treatments reaching patients across a more diverse set of modalities. While 50% where small molecules, 2023 saw the first approval for a CRISPR-based treatment.   Not only did we see further products introduced for rare diseases, but there is also a continued attraction of investors to this space. More than 1 billion USD was raised by biotechnology companies developing drug candidates with orphan drug designation further confirming the appetite from VCs into the field.   2023 also saw the foundation of Ampleia. Ampleia aims to be a central player in the rare disease space over the coming years and seeks the best technology and teams to create and support new companies with further products for rare diseases so that we can bring health equality for the 95% of rare disease patients who still do not have a cure. Rare Disease Day AFM-Téléthon

✨ [JOURNÉE INTERNATIONALE DES MALADIES RARES] 🌏 Dans le monde, plus de 300 millions de personnes, dont la plupart sont des enfants, vivent avec une des 7000 maladies rares. Savez-vous que 80% d’entre elles sont des maladies génétiques et près de 95% ne disposent pas de #traitements ? 🧬 À Genethon, plus de nos 200 #scientifiques et #experts sont mobilisés depuis la #recherche jusqu’aux essais cliniques pour concevoir et développer des #traitements de thérapie génique pour #guérir des patients atteints de maladies rares. Même si la thérapie génique a fait ses preuves, en remportant des premières victoires, l’accès à ces innovations thérapeutiques reste un obstacle. 🔬 A l’occasion de la Journée Internationale des Maladies Rares, nous mettons en lumière différentes #stratégies mises en place pour apporter des traitements de thérapie génique aux patients souffrant de maladies rares, telles que la #myopathie des ceintures, le syndrome de #Crigler Najjar ou encore la myopathie de #Duchenne. AFM-Téléthon Rare Disease Day Pour en savoir plus 👉 https://urlz.fr/pIDl #genethon #RareDiseaseDay #LightUpForRare #maladiesrares #journeemondialemaladierare #JIMR

At the occasion of the World Rare Disease Day, our COO Pierre-Albert Colcomb had a chance to share his views on the future and challenges of the field with students of the Chaire ESSEC Innovation et Santé. It was a good opportunity to tackle some received ideas on commercialization of programs for these diseases and illustrate how the biopharma industry can benefit from bringing treatments to patients affected by these diseases. Great discussions on patients access pathways, development incentives and market access for these patients in emerging countries! What a refreshing morning spent with the future leaders in the healthcare industry. Rare Disease Day AFM-Téléthon

Our COO, Pierre-Albert Colcomb, had the opportunity to present Ampleia's model to Takeda France team earlier this week and to discuss how we plan to support many more research projects in their translation toward clinical trial for rare diseases patients. Many thanks to BioLabs France and Christophe Tallec for this great opportunity and to Nienke Feenstra, Jean Delonca, Houda TAZI and the rest of Takeda's team for the nice discussions. #rarediseases #startupstudio #biotechnologies #venturecreation #TakedaFrance