The gene therapy market - France

As of 2020, the gene therapy market is undergoing significant growth and transformation. Globally, the regenerative medicine sector, which includes gene therapy, had over 987 companies with North America leading the way with 534 companies, a 10.3% increase from the previous year. Europe follows closely, with a particular dynamism in gene therapy, housing 237 companies, and a notable presence in the field of rare diseases. France stands out with 36 companies developing 100 programs across 84 unique gene therapy products. The market potential is considerable given the promises gene therapy holds for treating conditions such as cystic fibrosis, sickle cell disease, hemophilia, muscular dystrophy, and cancer. In 2019, the global gene therapy market saw record financing amounts, totaling $9.8 billion, with projections suggesting a growth to $13 billion by 2024, resonating a compound annual growth rate of 27.89%. Despite the high potential, the market confronts hurdles such as regulatory rigors, ethical considerations, and the risks associated with gene therapies. France's historical pioneering in gene therapy is challenged by administrative bottlenecks but is experiencing a resurgence owing to strategic government initiatives and investments, aiming to reclaim its leading position in the sector..Title: Navigating the Uncharted Waters of Gene Therapy Demand in France Gene therapy, an emerging frontier in the pharmaceutical industry, aims to cure rare and complex diseases by altering genes within diseased cells. France's quest to be a key player in gene therapy, especially in the realm of rare diseases previously neglected by laboratories, has seen a resurgence in recent years. The French market, rich in history as a pioneer in gene therapy, is today composed of 36 companies actively engaging in gene therapy and cellular development, showcasing a robust academic fabric featuring prominent research centers like Inserm, CNRS, and AP-HP. The demand for gene therapy in France is driven by the immense potential to address a myriad of diseases, including cystic fibrosis affecting between five and seven thousand people, sickle cell disease in around ten thousand individuals, hemophilia in seven thousand patients, muscular dystrophy impacting three thousand lives, and cancer, which saw more than three hundred and eighty-two thousand new cases in 2018. Notably, over thirty drugs are in the final phase of development, heralding a surge in potential treatments poised to revolutionize patient care. However, high development costs and strict safety protocols set by prominent regulatory bodies like FDA in the US and EMA in Europe keep the demand for gene therapy more potential than actual. Key risks and ethical considerations associated with gene transfer still loom large, tempering the pace at which these therapies enter the market. For France to maintain its competitive edge and avoid dependency on foreign therapies, considerable investments are paramount, along with fostering public-private partnerships. The French biotechnology value chain, despite its solid academic foundations and a roster of developing companies, contends with administrative hurdles that impede clinical trials, causing some firms like Cellectis to venture overseas for their research. As France stands at the crossroads of innovation and regulation, the future looks promising, with a projected growth of 84% from 2012 to 2019 in biotech research and development revenues. This indicates that while the gene therapy market in France is burgeoning, it's navigating through a crucial period of balancing breakthrough potential against the pragmatic realities of market and regulatory constraints..### Emerging Titans of Gene Therapy: A Glimpse into Pioneering Enterprises In the enthralling realm of gene therapy, a selection of formidable companies has emerged, traversing the complexities of biotechnological innovation and overcoming formidable challenges to pave the way for new horizons in healthcare. These companies are not mere participants but the prime movers in a field that promises to revolutionize the treatment of innumerable genetic conditions and diseases. **Cellectis: A Tale of Precision and Partnership** Originating from the illustrious halls of the Institut Pasteur, Cellectis stands out as a beacon of progress in the gene therapy domain. With an unwavering focus on targeted DNA modification, Cellectis has mastered the art of tailoring immune cells to combat cancer with finesse. In a symbiotic relationship, Pfizer, the pharmaceutical behemoth, anchors its confidence in Cellectis by owning a stake in its promise, with significant financial backing to bring innovative cancer therapies to fruition. With profound implications for immunotherapy, Cellectis's diligent use of TALEN technology, licensed from the University of Minnesota, manifests its prowess in a fiercely competitive landscape. **Genethon: The Vanguard of Therapeutic Revolution** As a torchbearer in gene therapy research, Genethon's credentials are accentuated by its inception under the patronage of the respected AFM-Téléthon organization. Leading the world in bioproduction capacity for gene therapy drugs, Genethon's state-of-the-art facilities underline its capability to bring groundbreaking treatments to life. Their collaborative ethos, forming alliances across various international borders, from the United States to Switzerland, underscores their commitment to delivering cures for rare genetic ailments. **Gensight Biologics: Illuminating Hope for Vision** Carving its niche in the gene therapy universe, Gensight Biologics is undeterred by the odds, particularly in its quest to preserve and restore vision. Founded in 2012, the enterprise has demonstrated tenacity by advancing two promising products through the alleyways of clinical trials. With a laser-like focus on ocular diseases, Gensight Biologics is sampling the future of gene therapy, making significant strides in treatments that light the way for the visually impaired. **Établissement Français du Sang (EFS): The Public Crusader** The EFS, triumphant in its quest for innovation, adeptly balances its roles as a key public organizational entity and a trailblazer in producing advanced therapeutic drugs. Being the foremost producer for clinical trial

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• Number of pages : 30 pages
• Format : Digital and PDF versions
• Last update : 01/09/2023

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Summary and extracts

List of charts

• Évolution des financements de la médecine régénérative par types
• Prospective de l'évolution du marché de la thérapie génique
• Nombre d'essais cliniques de médicaments de thérapie innovante par continent, initiés chaque année
• Évolution des financements de la médecine régénérative et de la sous catégorie : thérapie génique et thérapie cellulaire modifiée génétiquement
• Nombre d'entreprises spécialisées dans la médecine régénérative et les thérapies innovantes
• Répartition des essais cliniques (MTI) par phase
• Répartition des essais cliniques (MTI) européens par pays
• Répartition des programmes de R&D de thérapies génétiques
• Répartition des essais clinique (MTI) par catégorie
• Répartition des sociétés de développement de thérapie génique et cellulaire
• Les acteurs de la filière MTI
• Domaine d'expertise des acteurs de la filière MTI
• Évolution du chiffre d'affaires de la recherche et développement en biotechnologie
• Évolution de l'indice de chiffre d'affaires de la R&D en biotechnologie

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