Innovative therapies

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Genopole strengthens its support for biotherapies and health biomanufacturing

Genopole continues its commitment for the biomanufacturing of advanced therapy medicinal products by accompanying biocluster actors, facilitating dedicated conferences, participating in national actions for the support of the field, and consolidating its international partnerships for biotherapies and biodrugs in Europe and beyond.

A conference entitled "Innovative, Combinatory Therapies for Neuromuscular Diseases" was held at the biocluster on 16 May, in an in-person format to empower exchange. Organized by Genopole, in partnership with Professor Olivier Biondi from the Exercise Biology for Performance and Health Laboratory (LBEPS) and Doctor Émilie Vénéreau from the Tissue Regeneration and Homeostasis Unit at the San Raffaele Scientific Institute (Milan, Italy), the event drew more than 60 researchers in biotherapies and neuromuscular diseases. Presentations on pharmacological, cellular, genetic and physiotherapeutic innovations laid a foundation for a discussion on the promising perspective of combinatorial therapeutic strategies.

On 28 September, a hybrid colloquium entitled "Microbiota: Prevention and Health" attracted more than 150 participants. Organized in partnership with Inrae and the CNRGH, the event underlined the growing role of the microbiome in understanding and treating diseases. Two of the featured subjects were The Human Gut Microbiome Atlas and the French Gut Project, recently launched in France to analyze 100,000 fecal samples. Also, two Genopole businesses presented their research: Enterome, a company developing drug candidates from the intestinal microbiome; and Luxia Scientific, which is focusing on bacterial vaginosis diagnostics.

Genopole microbiota conference - Muriel Derrien, Danone Nutricia Research


On 7 April at Paris-Saclay, Genopole led the roundtable "The challenges of innovation in biomanufacturing: how to accelerate" at the sixth edition of the France Bioproduction Conference. The roundtable included Alain Lamproye (Yposkesi CEO), Angélique Manceau (Biowest sales director) José Castillo (Quantoom Biosciences CEO), Laurent Pépin (Genopole director of prospection & partnerships) and Serge Braun (AFM-Téléthon scientific director). The conference, organized by Polepharma and Medicen Paris Region (of which Genopole is a partner), united French and European actors in pharmaceutical biomanufacturing to discuss the sector's issues, potential synergies between digital sciences and biomanufacturing, and processes engineering.

Genopole is giving impetus to health biomanufacturing in France within its role as a founding member of France BioLead. Officially launched on 7 December by the French Ministry of Economics, Finance and Industrial and Digital Sovereignty, France BioLead associates 15 public and private entities with the mission of structuring, piloting and promoting the nation's biodrug manufacturing sector and its actors. Notably, one of its primary objectives is to double biodrug manufacturing in France by 2030.

Internationally, Genopole was chosen by the European Union to coordinate the COBIOE project, a European alliance for health biomanufacturing (see page 25).


The Évry ecosystem commits to the Biodrugs Major Challenge

On 3 February 2022, the French Government's Biodrugs Major Challenge action (a part of France 2030), with its budget of €13 million, retained eight novel technological projects for the production of innovative bio, gene or cell therapies aimed at rare or common diseases. The competencies of four Genopole entities, the laboratories ART-TG, CiTHERA and I-Stem and the company Yposkesi, will be called upon for three of the laureate projects:

  • ASMA (Acoustic solutions for manufacturing advanced therapies) combines technologies to improve the key steps of gene material transfer and thus production yield in gene therapies.
  • STELLAR (Stem cell differentiation evaluation on line–light holographic analysis recording) aims to be a continuous and non-destructive quality control system for cell therapies.
  • SOFTCELL is taking inspiration from phytoplankton culturing to develop a bioreactor for the high-volume production of human stem cells.
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L’écosystème évryen engagé dans le Grand Défi Biomédicaments

Seven years of hindsight and conclusive results for a Genethon gene therapy

Gene therapies represent the main source of hope for people with Wiskott-Aldrich syndrome, a severe immune deficiency caused by mutations in the WAS gene, leading in turn to defective white blood cells. The laboratory Genethon conceived and produced a lentiviral vector and promoted an international ex vivo gene therapy clinical trial aimed at this disease. An article published 24 January in Nature Medicine provided long-term efficacy results after a mean seven years of follow-up of eight patients aged 8 months to 30 years at the time of treatment. Its authors reported the resolution of primary symptoms such as recurrent infections, a reduction in bleeding and autoimmune disorders, and no observed adverse effects.

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Des résultats concluants pour une thérapie génique de Généthon

Enabling CAR T-cells for solid tumors

The SysFate team at the Genomics Metabolics Unit is participating in the European project T-FITNESS chosen by the European Innovation Council and launched on 1 September to make CAR-T cell treatments for solid tumors a reality. CAR T-cell therapy is a type of immunotherapy that harnesses the immune system's T lymphocytes for the battle against cancer. Although remarkably efficacious against blood cancers, this approach has, to date, shown only limited action against solid cancers. Hoping to change this latter aspect, the Genopole team will, in a first step, use its genome analysis expertise and TETRAMER bioinformatic tool to identify the transcription factors responsible for the loss of efficacy of CAR-T approaches over time.

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Vers un nouveau traitement des tumeurs solides ?

Genethon: uncovering a second key mechanism of Duchenne muscular dystrophy

Genethon's Progressive Muscular Dystrophies team has identified a second source of muscle dysregulation in Duchenne muscular dystrophy (DMD) involving mitochondria, the cellular organelles responsible for energy production.

Since the 1970s, muscle energy loss in DMD was attributed uniquely to disturbed calcium regulation. The Genethon team's work has indeed brought to light a more complex supplementary mechanism involving the dysregulation of microRNA expression directly affecting mitochondrial activity and leading to the degeneration of muscle fibers. This notable breakthrough puts novel molecular pathways on the map and thus creates new therapeutic possibilities to explore.

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Myopathie de Duchenne

An artificial intelligence that explains its choices

Researchers from the Computer Science, Bio-informatics and Complex Systems (IBISC) laboratory have developed an artificial intelligence (AI) that furnishes clinicians with a medical diagnosis and the explanatory biological data to back it up. Called GraphGONet, the system is built upon a new type of neural network integrating knowledge on genes, their expression levels, the proteins and associated biological functions they code for and more, in its deeper layers.

The user is provided with information on the biological elements involved and how much each contributes to the AI's diagnosis. By associating prediction and explanation, GraphGONet respects the need for an IA to be transparent, which ensures the clinician as to the validity of the medical diagnosis. Such a tool will contribute to the growth of personalized medicine and help unveil mechanisms involved in specific diseases.


A therapeutic advance for three genetic diseases

Researchers from I-Stem and Genethon, in partnership with the AI company Kantify, have identified a promising drug combination in the setting of three genetic diseases sharing similar molecular mechanisms. In a cellular model of limb-girdle muscular dystrophy type R3 (LGMD-R3), the team tested nearly 1,000 therapeutics already approved by the American Food and Drug Administration for other indications. Alpha-sarcoglycan is a striated muscle protein that, although misfolded in people with LGMD-R3, does retain some of its function—if it is not broken down by cellular quality control systems. Thus the team's objective was to identify compounds able to block that protein breakdown. Aggregating results from cellular tests and data on the available drugs, the team's AI-driven approach identified therapeutic potential in 47 compounds. Therein, the researchers identified moreover an association of two compounds showing a strong capacity to inhibit the breakdown of muted α-sarcoglycan. In their work, the researchers also showed that this protein breakdown involved not one but two mechanisms, an observation that allowed them to enlarge their results to two other diseases sharing traits with LGMD-R3: limb-girdle muscular dystrophy type R5 and cystic fibrosis.

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Une piste thérapeutique contre 3 maladies génétiques

Olivier Biondi explores mechanisms activated by physical exercise

Laureate of the 2020 Atige, Olivier Biondi is now making his projects real at the biocluster. Recently named as a professor at the University of Évry-Paris Saclay, he created his team within the Exercise Biology for Performance and Health Laboratory (LBEPS) and began his pioneering research on the effects of physical exercise on neuromuscular diseases.

In the medium term, Olivier Biondi aims to furnish physicians with scientific data enabling the adaptation of exercise to each patient and each disease. Further in the future, he will study the synergistic effects of exercise when combined with pharmacological, gene, cell, or other therapies.

We are developing, on one hand, protocols to have an extensive view of the mechanisms of action of physical exercise, and on the other, more precise cellular and molecular analyses to better understand those activated mechanisms.
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Les mécanismes activés par l’exercice physique

A trio of genome sites associated with arterial hypertension

A large-scale genomics study published in Nature Communications has unprecedentedly revealed three genome sites associated with primary hyperaldosteronism, a leading cause of arterial hypertension. That work called upon the competencies in genomics and mathematics/bioinformatics available at the National Center of Human Genomics Research (CNRGH, CEA/François Jacob Institute of Biology) and the Paris-Cardiovascular Research Center (PARCC). The discovery of more frequent variations at those sites in people with primary hyperaldosteronism may ultimately improve early detection of the disease and bring a better understanding of the mechanisms involved in it.

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Trois sites génomiques

Enalees sells its human diagnostic branch to Biosynex

In 2020 and in partnership with Bertin Technologies, Enalees developed a rapid isothermal amplification SARS-CoV-2 RNA detection test on a portable reader validated with the help of Institut Pasteur. Enalees is now selling that human diagnostic branch to the Alsatian company Biosynex.

"Enalees will continue to produce its human tests for Biosynex for one year," states company CEO Laurent Thiery. "In parallel, we will pursue the development and production of tests for infectious diseases in animals, which is the focus of our company." In other news, Enalees obtained a €500,000 grant from the Île-de-France Administrative Region as part of the France 2030 investment plan to solidify its supply chain, develop its range of products and enlarge its facilities at Genopole.

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Trois sites génomiques

A European boost for SynHelix

Created in the Shaker, welcomed to Genopole and developed in the Booster, SynHelix has a solid automated technology for the synthesis of long DNA fragments. Its disruptive innovation, a less costly alternative to other existing processes, has the notable advantage of applicability in numerous markets, and particularly in health. The Belgian Group Univercells, a specialist in biodrugs has acquired SynHelix and made it its French affiliate under the new name of Quantoom Biosciences. The company continues to pursue its development with Genopole, assures its CEO Irina Gbalou: "We intend to stay in Évry-Courcouronnes, to maintain our ties to Genopole's rich ecosystem and its great perspectives for recruitment, projects and partnerships."

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Innovhem recognized for its sickle-cell anemia management method

The yearly Créatrices d'Avenir Trophies recognize women heading innovative Île-de-France companies. In 2022, its Innovation Trophy went to Marie Cambot, CEO and cofounder of Innovhem, which was also one of four French companies chosen by the European Union's Women TechEU program.

Innovhem, a company born in the Shaker and accelerated in the Booster, is developing a medical system to improve the management of sickle-cell anemia, the world's most common genetic disease. The company's innovation combines disease biomarkers and artificial intelligence, and "responds to a true need," explains Marie Cambot. "A survey of physicians revealed that 90% of them consider that they do not have sufficient diagnostic tools available to them," she adds. "However, precisely defining disease severity and predicting its course are vital to the physician's ability to propose personalized treatments to each patient."