Ampleia

🎄✨ As we are wrapping-up the last few days of 2024, Ampleia's team reflected on key progress made this year and drew perspectives for 2025. 🚀 Key progress: - Growth of our deal-flow reaching 240 projects in orphan drugs (excl. oncology) scouted since inception. This reflects Ampleia's capabilities to meet with world-class research scientists from leading institutions, promising biotechs entrepreneurs and pharma partners to build future champions in their areas. - Team expansion with the appointment of Ralph Laufer as Advisor and Permanent Member of our Investment Committee. Ralph joins Braun Serge, Jean-François BRIAND, altogether totaling a century of experience in orphan drugs, research and pharma development. - Incubation of projects with the launch of a first NewCo by Q1-25. More to be announced in 2025! 📈 Perspectives of the industry for 2025 TAs and technology modalities of interest: Our deal-flow review at 2024 closing showcases that CNS remains of strong interest for research institutions with >20% of the programs we received addressing indication in that area. While neuromuscular ranks 2nd, we experienced a jump in in autoimmune and renal disorders, fueled by the appetite from pharma for these diseases. Small molecules will definitely be leading the trend in 2025 (~35% of our deal-flow) even though researchers continue to push gene therapy approach which correlates with the fact that 80% of rare disorders have a genetic origin. Go more entrepreneurial: We continue to see more pharma development executives pivoting from pharma to VCs and bringing their incredible translational experience to serve early-stage projects and nascent technologies. We understand that trend as fueled by a growing need from VCs to mitigate risks in translational phase and streamline development in line with pharma expectations, leveraging know-how developed by big pharma to do exactly that. In parallel, growing AUM have made possible these costly hirings, opening more opportunities for pharma executive profiles to turn to the VC world. We are all excited by the opportunities and promises of 2025 and we look forward to collaborating with exceptional researchers, entrepreneurs and partners in 2025! Enjoy the Holiday Season! ✨🎄 Emilienne Soma Marion Pilorge, Ph.D, Ulku Cuhadar, PhD, MBA, Pierre-Albert Colcomb Alexandre Lemoalle #venturebuilder #orphandrugs #lifescience

🌟 Exciting News from BioEurope! 🌟 Ampleia team had an enriching experience at BioEurope last week! 🎉 We had the opportunity to meet with a diverse range of partners, from innovative biotech companies to venture capital firms, leading pharmaceutical giants and top universities. The event was a fantastic platform for networking, collaboration, and learning. We engaged in insightful discussions and explored potential collaborations. It was a truly enriching experience that has opened up new avenues for growth and innovation. A big thank you to all the organizers and participants for making BioEurope such a success! We look forward to continuing these conversations and building strong partnerships in the future. Marion Pilorge, Ph.D Ulku Cuhadar, PhD, MBA #BioEurope #Biotech #Pharma #Innovation #Networking #Collaboration #Teamwork #Success

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