BUILDING SECTORS

BUILDING SECTORS

From its roots in the development of innovative therapies, Genopole is expanding into the bioeconomy, a growing sector at the biocluster, and computational genomics, an emerging sector that is an enabler of biotech applications. These sectors are being adopted as a biocluster development orientation because they are in harmony with Genopole’s mission, which is to support the entire innovation maturation chain, from targeted training to basic and applied research and on to industrial development.

Genopole: a major actor in innovative biotherapies

Genopole is one of the world’s leading sites for the development of innovative therapies. Renowned for its scientific excellence, the Essonne biocluster is home to six laboratories in the field, two of which are pioneers in France: Genethon, a specialist in gene therapies for rare genetic diseases; and I-Stem, a specialist in the use of human pluripotent stem cells for pathological modeling, drug candidate screening and cell therapy development. Confirming the predicted arrival of the first clinical trial successes and market launches for innovative therapies, 2020 was remarkable for its advances, notably for gene therapies and again therein for Duchenne muscular dystrophy, the emblematic symbol of AFM-Téléthon’s commitment since its inception 30 years ago.

Building a national biotherapeutics sector

Genopole unites numerous assets to accompany the biotherapeutics revolution and respond to its scientific, technological, economic and industrial challenges, as was underlined in a note on innovative biotherapies in Île-de-France published in 2020 by Institut Paris Region. Genopole’s role in the construction of a national sector for health biotechnologies and innovative biotherapies was also illustrated by the recognition of a team of two biocluster actors as an Industrial Integrator within the Biopharmaceuticals category of the French Government’s Major Challenges program.

The biocluster offers all the links in the chain: initial and continuing training, academic research laboratories, R&D and services companies, a technology transfer accelerator and production centers for clinical batches and medicines, all of which benefit from Genopole’s teams, support services and technological equipment.

The biotherapies sector at Genopole

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WEBINAR: ADVANCES IN GENE THERAPY

On 8 October, in partnership with Choose Paris Region, an agency fostering the international attractiveness of the Île-de-France Administrative Region, Genopole held a webinar on gene therapies. Intended to attract the attention of North American companies and more specifically CROs et CDMOs in the gene therapy sector, the live webinar united about a hundred foreign participants with Genopole’s bioproduction actors: ART-TG, Yposkesi, Texcell and WhiteLab Genomics.

ÎLE DE FRANCE AT THE CUTTING-EDGE OF INNOVATIVE BIOTHERAPIES

Genopole contributed to a note published by Institut Paris Region to promote the biotech and gene & cell therapy know-how available in Île-de-France. The document maps the region’s primary academic and industrial actors in next-generation biopharmaceuticals research. Genopole is a prominent feature in that landscape: with its dynamism throughout the value chain, from training to R&D and through to bioproduction, the biocluster is contributing to the construction of an innovative biotherapies sector in France.

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RESEARCH

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A symbolic advance in the fight against myopathies

An important milestone was reached in 2020 by Genethon, a major actor in innovative biotherapies at Genopole, created 30 years ago by AFM-Téléthon.
Indeed the ANSM, the French national agency for medicines and health products safety, authorized Genethon’s clinical trial on Duchenne muscular dystrophy (DMD), a rare genetic disease emblematic of AFM-Téléthon’s mission. The trial, developed with Sarepta Therapeutics, will evaluate the tolerability and efficacy of an innovative gene therapy conceived by the laboratory. That therapy involves the delivery of a shortened but nonetheless functional version of the gene coding for dystrophin by an AAV vector. The dystrophin protein is essential to muscle function, but altered in DMD. The trial will be started in several French centers and expanded internationally thereafter.

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COMPANIES

NEW SUCCESS FOR A GENETIC IMMUNODEFICIENCY DISORDER

Two of Genopole’s major actors in biotherapies, the laboratory Genethon and the gene therapy manufacturing facility Yposkesi, contributed substantially to a new success in gene therapies for a severe immunodeficiency. They developed a lentiviral vector for a novel gene therapy aimed at providing lasting treatment for chronic granulomatous disease and produced drug batches for an international trial to test the medication. Of the nine patients who received this latter, seven experienced no infections and six were freed from treatments for disease complications.

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CRISPR-CAS9 FOR BLOOD AND METABOLIC DISEASES

A team from the Integrare laboratory developed an innovative approach for the treatment of metabolic disorders and genetic blood diseases such as hemophilia. The approach calls upon the precision of the “molecular scissors” CRISPR-Cas9 to modify hematopoietic stem cells (which give rise to all blood cell types) in such a way as to produce therapeutic proteins in the erythroid lineage, i.e., that giving rise to red blood cells, by far the most numerous cells in blood. The proteins secreted by these cells are captured by the diseased cells wherein they are able to correct metabolic disorders.

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A STEP CLOSER TO CONTROLLING RISKS IN GENE THERAPY

Immune responses against a viral vector carrying a gene therapy can create risks for that treatment, including the possibility of rendering it ineffective. Researchers from Genethon, in partnership with CNRS/Inserm and Spark Therapeutics teams, successfully inhibited immune responses to AAV vectors. Their results open new therapeutic vistas, for example, the ability to role out gene therapies to a larger population of patients or to enable their repeated administration over time.

COMPANIES

AN EARLY TEST FOR ALZHEIMER’S DISEASE

AgenT was chosen for Challenges Magazine’s list of 100 start-ups to invest in 2020. A laureate of the second edition of Genopole’s Booster program, AgenT is developing a blood test and an AI algorithm to detect Alzheimer’s disease up to 20 years before any symptoms appear.

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COMPANIES

SYNERGIES FOR GENE THERAPY INDUSTRIALIZATION

By joining their forces, Genopole’s companies and labs are rising to the difficult challenge of industrializing the production of advanced therapy medicinal products, first for clinical then for commercial batches. Here’s an example among Genopole entities: PTG1+ is a polymer developed by Polytheragene to improve the bioproduction of gene therapy vectors. That polymer is an essential component in a production upscaling protocol developed by Yposkesi. And finally, Genethon will deploy Polytheragene’s product and Yposkesi’s protocol in clinical trials for gene therapies aimed at neuromuscular disorders and other rare diseases.

COMPANIES

AB Science pursuing its tests on masitinib in several disease settings

The Euronext-traded company AB Science and its laboratory at Genopole are carrying out several clinical trials to test their product masitinib in a range of pathologies including multiple sclerosis (MS). Results obtained in the two progressive forms of MS have been encouraging. “Masitinib should improve the lives of patients with MS, with fewer relapses, slowed decline, and more time before needing a wheelchair, and perhaps not needing one at all,” comments Laurent Gros, head of AB Science’s Drug Discovery Program at Genopole.

Partnerships with the United States

The tyrosine kinase inhibitor masitinib was also identified by researchers at the University of Chicago as the most powerful inhibitor of the principal protease of SARS-CoV-2, specifically the protease needed for viral replication in COVID-19. “The American researchers contacted us to optimize their protocol. We’ve laid the foundation for a potential partnership concerning an antiviral indication for masitinib against COVID-19.” For its part, AB Science is carrying out a clinical trial to test the ability of masitinib to control the excessive inflammation brought on by an overly-strong immune response in COVID-19. The potential antiviral+anti-inflammatory action of masitinib inspired a €15 million loan from the European Investment Bank for the biotech to enable the pursuit of clinical development. Other trials have looked at the performance of masitinib in such settings as severe asthma, amyotrophic lateral sclerosis, pancreatic cancer and Alzheimer’s disease.

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RESEARCH

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SKIN STEM CELLS AND RADIOSENSITIVITY

The researchers at the Genomics and Radiobiology of Keratinopoiesis Laboratory (LGRK) are specialists in human skin stem cells. They study factors that regulate stemness, with such objectives as improving the regenerative potential of grafts for cell therapies. They also characterize mechanisms that maintain or disturb genome stability. For example, in 2020, the LGRK team showed that small doses of radiation, like those used in medical imaging, although safe for healthy skin, can alter actively regenerating cells. The lab also identified a new molecular signature of radiosensitivity in patients who have severe adverse effects after radiation therapy.

RESEARCH

Biodrugs: Magenta receives French industrial integrator label

The Magenta project, which unites the Genomic Therapy Technology Research Accelerator (ART-TG, Inserm) headed by Anne Galy with the CiTHERA cell therapy lab (Inserm, University of Évry, Paris-Saclay University) headed by Annelise Bennaceur-Griscelli, received the Industrial Integrator label within the Biopharmaceuticals category of the French Government’s Major Challenges program. Genopole contributed to equipment acquisition and provided an Atige grant to ART-TG so that it could develop its R&D programs in the best possible conditions.
The objective of the Biopharmaceuticals Major Challenge is to improve yields and costs for the production of biodrugs in France. The two labs are both located within Genopole’s Cytopolis building, which is dedicated to the development of innovative biotherapies and their rapid advancement to the patient’s bedside.

An intersection for industrials, start-ups and laboratories

Magenta is one of six major actors in the sector having received the Industrial Integrator label from the French State’s Innovation Council. They all share the mission of providing project holders with the competencies and tools needed for the production of antibodies, cell therapies and gene therapies & their vectors.

Magenta will also be an intersection at which industrials (pharmaceutical companies, equipment suppliers, etc.) seeking novel production technologies can meet with the innovative companies and academic laboratories forwarding such an offer. It will furthermore provide a fruitful environment for the development of start-ups born of the research promoted within the Integrators.

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