Summary of our market study

The gene therapy market is experiencing significant growth.

worldwide, the regenerative medicine sector, which includes gene therapy, numbered over 1,000 companies. North America leads the way with over 530 companies, followed by Europe with over 230.

The global gene therapy market is estimated at over $7 billion. It is attracting record amounts of funding. This figure is set to rise to $13 billion by 2024.

France is a historic pioneer in gene therapy

Demand for gene therapy in France

Gene therapy aims to cure rare and complex diseases by modifying genes in diseased cells.

France is seeking to become a key player in gene therapy, particularly in the field of rare diseases neglected by laboratories.
Demand for gene therapy in France is stimulated by the immense treatment potential of numerous diseases, including cystic fibrosis, which affects between five and seven thousand people, sickle cell anemia, which affects around ten thousand individuals, hemophilia, which affects seven thousand patients, muscular dystrophy, which affects three thousand people, and cancer, which registers over 380,000 cases per year.


France has more than 36 companies developing 100 programs for 84 unique gene therapy products. These companies are active in gene therapy and cell development in association with leading research centers such as Inserm, CNRS and AP-HP. More than thirty drugs in the final stages of development

High development costs and stringent safety protocols established by major regulatory bodies such as the FDA in the US and the EMA in the UK, have led to the development of more than 30 new products.and the EMA in Europe, the risks and ethical considerations associated with gene transfer remain significant, and are slowing the pace at which these therapies enter the market.

Gene therapy players

Cellectis: A spin-off from the Institut Pasteur, Cellectis focuses on targeted DNA modification. Pfizer finances and holds a stake in the company, which uses TALEN technology licensed from the University of Minnesota.

Généthon: World leader in the biomanufacturing of gene therapy drugs.

Gensight Biologics: Founded in 2012, the company focuses on eye diseases,

Établissement Français du Sang (EFS): a pioneer in the production of advanced therapeutic drugs and the world's leading producer of clinical trials

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Summary and extracts

1 Market overview

1.1 Definition and scope of study

The therapy gene has a simple goal, to cure rare diseases by changing genes. The principle of gene therapy consists of inserting modified genes via a vector into a diseased cell in order to modify its functioning. The reality is more complex and the processes are multiple. Its aim is to deal mainly with genetic diseases, but also with new diseases, cancers and cardiovascular diseases.

Gene therapy drugs are part of the MTI (advanced therapy medicinal products) is a medicinal product manufactured from cells, or human genetic material, to treat cellular or genetic abnormalities.

The market demands investments colossal for results that are still very random. Although a first drug based on gene therapy was first developed in China in 2003, it was not until 2011 that a Dutch company became the first to offer such a drug in the West. On the other hand, the development possibilities are very promising because research is progressing. The United States is unquestionably the market leader, with some 15 companies, each listed at several billion dollars. Indeed, in a sector where investment is crucial, the major resources deployed by the United States give it a leading position

In the area of rare diseases gene therapy is relatively new. The first trials only date back to the 1990s. For a long time, these diseases did not arouse the interest of laboratories because they affect only 5 people out of 10,000 . Today, the stakes are high and many laboratories are working on these issues. Thus, competition is becoming more structured, but still remains weak because of the numerous fields of application and ethical controls.

A former pioneer with the pastoral institute, the France is a promising country of gene therapy in Europe. Various ITMs are currently being developed by companies such as Cellectis, Genethon, Gensight and Horama in private or public production sites (AP-HP, EFS). Today, France seems to be back in the race: the State has emphasized the importance of regaining a leading position in the sector, because the country that develops an ITM, has a good chance of producing it and therefore putting it on the market. The risk for France is that it will not be able to develop its ITNs quickly enough, and that in a few years' time it will be dependent on ITNs from the United States.

Numerous research labs are therefore publicly funded . In the absence of direct economic spin-offs, there are few companies and public-private synergies are frequent. The first treatment marketed in Europe by Uniqure has an estimated cost per patient of more than one million euros. Faced with such a sum, research and human tests can only be financed by governments, or by partnerships with private companies (often foreign)

List of charts presented in this market study

  • 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
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Latest news

Sanofi plans to spin off its Consumer Healthcare business in 2024 - 28/10/2023
  • Year of separate listing of Sanofi's Consumer Healthcare Division: 2024
  • Sanofi envisages savings of: up to 2 billion euros between 2024 and the end of 2025
  • Presence of Sanofi Consumer Healthcare Division: 150 countries, more than 11,000 employees
Sanofi to spin off its consumer healthcare division and float it on the stock market - 27/10/2023
  • Sanofi is a French pharmaceutical company
  • The Consumer Healthcare division has over 11,000 employees in 150 countries.
  • The demerger of Consumer Healthcare will take place in the fourth quarter of 2024 at the earliest.
  • This division accounts for just over 10% of Group sales.
  • In 2022, the Consumer Healthcare division generated 5.1 billion euros of Sanofi's 43 billion euros in sales.
  • The Consumer Healthcare division posted growth of 13.7% in 2022.
  • By 2021, the global consumer healthcare market will be worth over €180 billion.
  • The market share of Haleon, the world leader in consumer healthcare, is estimated at 6% in 2021.
  • The over-the-counter segment accounts for 90 billion euros, vitamins, minerals and supplements for 50 billion euros, and oral care for almost 30 billion euros.
  • Its Dupixent sales approach 11 billion euros in annual sales.
  • Beyfortus vaccine is currently marketed in 4 countries, including France.
Sanofi inaugurates Europe's largest vaccine plant at Val-de-Reuil - 07/09/2023
  • Sanofi's vaccine plant in Val-de-Reuil celebrated its 50th anniversary.
  • Sanofi has invested 250 million euros in two modernization/expansion projects at the plant, which employs 2,200 people.
  • It ships 600 million doses of vaccines worldwide every year.
  • Sanofi has set up a new influenza vaccine production unit, at a cost of 200 million euros.
  • Over the past ten years, Sanofi has invested 600 million euros in Val-de-Reuil.
  • Sanofi plans to double sales of its vaccines division between 2018 and 2030, to more than 10 billion euros a year.
Sanofi invests $250 million in flu vaccines in France | Sanofi invests $250 million in flu vaccines in France - 07/09/2023
  • Sanofi is the world's fourth-largest vaccines company
  • Sanofi invests 250 million euros in its Val-de-Reuil site, Europe's largest vaccine production facility
  • Sanofi has 11 vaccine plants
  • 50% of Sanofi's production is concentrated in influenza.
  • Sanofi increases influenza vaccine production capacity by 40%
  • Sanofi's Efluelda vaccine is recommended for the elderly and launched in 18 countries, including 11 where it has been recommended by health authorities.
  • Sanofi has invested over 600 million euros in its Val-de-Reuil site over the past ten years.
Sanofi gains a foothold in the obesity market with biotech Adocia - 10/07/2023
  • Appetite suppressants derived from antidiabetics are an emerging market
  • . Industry giants such as American Lilly and Danish Novo Nordisk are already involved in this market.
  • Sanofi paid 10 million for the exclusive right to negotiate a worldwide partnership for Adocia's M1Pram rapid insulin.
  • Adocia's M1Pram rapid insulin is special in that it makes you lose weight instead of gaining it.
  • The potential market for appetite suppressants derived from antidiabetics is estimated at several tens of billions of dollars a year.
  • Sanofi is a specialist in type 1 diabetes, and M1Pram could benefit obese patients suffering from this type of diabetes - around 5% of type 2 diabetics.
  • Phase 2 clinical results for M1Pram showed exceptional weight loss with good glycemic control in overweight and obese type 1 diabetics.
  • The population that could benefit from M1Pram is estimated at nearly 40 million people worldwide, representing a potential market worth several billion dollars.
Sanofi finally has its Covid-19 vaccine - 13/11/2022
  • Doses ready for distribution
  • The French laboratory has accepted the dominance of the RNA vaccine and the American Moderna and German BioNTech
  • The recombinant protein technology used in its VidPrevtyn Beta will make it possible to vaccinate populations in regions where the cold chain is difficult to maintain

Companies quoted in this study

This study contains a complete overview of the companies in the market, with the latest figures and news for each company. :

Sanofi Aventis
Cellectis
Généthon
Gensight Biologics

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