Innovative therapies

Genopole: a motor for biotherapies

In 2021, Genopole strengthened its place as a leader in biotherapy development and production. Forty laboratories and businesses, with the support of eight technological platforms, contributed to the discovery, optimization and clinical & industrial development of gene, cell and other innovative therapies.

By providing equipment and recruitment assistance, Genopole lent its support to the growth of the Genomic Therapy Technology Research Accelerator (ART-TG) and the Cithera innovation center. The mission of this latter is the onsite construction of a clinical-grade stem-cell bank for cell therapies. Together, ART-TG and Cithera form the Magenta industrial integrator as accredited by the Biopharmaceuticals Major.

Challenge. Genopole presented Magenta in Institut Paris Région’s September 2021 Note Rapide, which provided an overview of Île-de- France actors in biotherapies & biomanufacturing and the issues in those fields.

Genopole also strengthened its commitment to the regional and national dynamic for health biomanufacturing set in motion in 2020. With 11 other clusters and competitiveness hubs, Genopole participated in the French Bioproduction Tour 2.0, and organized its third of May Île-de-France stop. That initiative strengthened bonds between the regional actors and stimulated the creation of a national structure for health biomanufacturing.

Year 2021 witnessed the first regional conference for biotherapies and biomanufacturing, headed by the Île-de-France Region and in partnership with Genopole, the Medicen cluster, and Choose Paris Region. That event invited 130 participants from academia, politics, industries and start-ups to debate propositions aimed at strengthening biomanufacturing in Île-de-France. It resulted in 11 recommendations to structure the biotherapy ecosystem, increase the region’s attractiveness and accelerate biomanufacturing industrialization.

The France Biomanufacturing Congress, a major yearly event for the sector, included a roundtable held 23 June and facilitated by Genopole on the subject of accelerating novel biomanufacturing means. Three Genopole biotech start-ups, Fabmid, Synhelix and Core Biogenesis, presented the potential of their innovations to improve the yield and quality of processes while reducing costs.

"The Biotechnological Revolution and Tomorrow’s Medicine"

Genopole wrote a chapter on gene therapies for the book "La révolution biotechnologique et la médecine de demain" (The Biotechnological Revolution and Tomorrow’s Medicine) published by Doin Editions on 18 March 2021. Intended primarily for students and teachers, the work presents the technologies derived from life sciences that are currently changing patient care and laying a path to personalized medicine in cancers, genetic diseases, viral diseases, metabolic disorders, bone diseases and much more.

Yposkesi extends its biomanufacturing capacities

One of Europe’s largest developers and manufacturers of viral vectors for gene therapies, Yposkesi proposes its know-how to biotechs and pharmaceutical companies active in the research or marketing of advanced therapy medicinal products (ATMPs). To increase its biomanufacturing capacities, Yposkesi is building a new 5,000 m² plant at Genopole. Once active, it will be one of Europe’s largest sites dedicated to ATMPs. The site represents an investment of €58 million, of which €1.3 million is covered by a subvention from the France Relance call for proposals. In the past, Genopole had contributed €8 million to finance the construction of Genethon Bioprod, which, in 2016, became Yposkesi and today part of the South Korean group SK Pharmteco.

"This extension demonstrates our exceptional technological expertise in the biomanufacturing of gene therapies."

Alain Lamproye, Executive President of Yposkesi

5,000 m²

of floorspace for Yposkesi’s second site, one of Europe’s largest for advanced therapy medicinal products.

Yposkesi extends its biomanufacturing capacities

Pep-Therapy tests its cancer drug candidate

Pep-Therapy develops innovative, bifunctional "cell penetrating & interfering" peptides, that is, a combination of small proteins able to enter into cells and thereafter specifically block key protein-protein interactions involved in pathologies while leaving normal mechanisms untouched. After raising €2.75 million, the company teamed with Institut Curie to launch the first clinical trial for its drug candidate PEP-010 in the setting of solid tumors and more particularly in metastatic triple-negative breast cancer and platinum-resistant ovarian cancer. Preliminary data on safety and tolerability are expected for 2022.

Duchenne muscular dystrophy may appear before birth

The Muscular Diseases team at I-Stem performed a study to differentiate iPS cells into muscle cells and analyze the expressed genes. In so doing, the researchers were able to reveal very early manifestations of Duchenne muscular dystrophy. Indeed, molecular dysregulations are observed in the cells of patients with the disease compared to those of healthy controls. The study’s results suggest that the disease may develop before birth and even before myogenesis, and thus also point to a need for novel consideration of the myopathy’s course, as its belated diagnosis, usually around the age of four, could potentially limit treatment efficacy.

AML: AB Science launches clinical studies

AB Science, which has a laboratory at Genopole, has been authorized by the ANSM, France’s drug safety authority, to begin phase I/II clinical study of its anticancer molecule AB8939 in the setting of acute myeloid leukemia (AML), a quick-spreading hematological cancer that develops in the bone marrow and spreads rapidly to the blood. The trial will evaluate the tolerability and efficacy of the molecule as second and third-line treatments in patients who are not able to undergo intensive chemotherapy. The same authorization was also accorded to AB Science by the Canadian health authority.

Three simultaneous publications on major diseases for SABNP

The original method developed by the laboratory SABNP (structure and activity of normal and pathological biomolecules) to study protein-RNA interactions is proving its aptitude for discoveries. The method combines the lab’s patented technology MicroTubule Bench with high-resolution imaging acquired in 2021 thanks to support from Genopole and the University of Évry. The simultaneous publication of three manuscripts in September illustrates the reach of their approach: working with academic teams and the Genopole company Synsight, the lab was able to elucidate three key mechanisms in cancers and neurodegenerative diseases, and identify a potential drug treatment.

Treating deep wounds with fish skin

CERITD is sponsoring a clinical trial on the use of therapeutically-prepared fish skin for the treatment of deep diabetic ulcers. The trial is coordinated in France by the Diabetic Foot Unit at the South Île-de-France Medical Center, a Genopole partner. The healing and infection-prevention effects of the fully-natural, omega-3-rich fish-skin matrix will be tested on more than 200 patients.

15 genetic risk factors for Lou Gehrig’s disease

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, causes motor neuron death and progressive paralysis of all the muscles. Genethon’s DNA and cell bank contributed to the largest ever pangenome-wide association study on this currently untreatable and ultimately fatal disease. Using DNA samples from more than 30,000 patients and more than 120,000 controls, the scientists searched for variations frequently associated with the disease across the entirety of the genome. They identified 15 such sequences correlated to increased ALS risk. Those sequences took the form of genes, repeated sequences and regulatory elements for which further studies may elucidate involved mechanisms and shed light on possible therapies.

Promising gene therapy for Charcot-Marie-Tooth disease

Nicolas Tricaud heads the Diseases of Myelinated Fibers team, created at I-Stem through the support of the Genopole Atige program. He has published promising results from a gene therapy study performed in an rat model of Charcot-Marie-Tooth disease type 1A. This latter is a hereditary neurological disorder resulting from damage to the myelin sheaths of peripheral nerves. In the study, the adenoviral vector carrying the gene therapy was injected directly into the sciatic nerves of the rats, targeting thus the cells affected by the disease. The therapy greatly reduced myelin sheath defects and motor and sensory deficiencies.

"The Genopole concept saw the day in the labs of AFM-Téléthon. Its history and identity are inseparable from the development of genomics and its application to human health, and resultantly the South Essonne area has become a major actor in tomorrow’s medicine"

Christian Cottet, CEO of AFM-Téléthon

Promising gene therapy for Charcot-Marie-Tooth disease