The cell and gene therapy market was valued at USD 6 billion in 2017 and is projected to exceed USD 35 billion in value by 2026. Moreover, these therapies stand to redefine medical paradigms by providing treatments for previously incurable diseases and usher in a new era of precise and individualised therapies. Here we showcase five of the key companies working in next-generation cell and gene therapies in Europe today.
Euronext Amsterdam and Brussels: KDS
CEO: Arthur Lahr
Headquartered in Amsterdam, the Netherlands, novel cell therapy firm Kiadis harnesses the power of natural killer (NK) cells to develop cancer immunotherapies. The Kiadis pipeline includes products which evaluate NK cells in post-transplant patients with acute myeloid leukaemia and myelodysplastic syndromes as well as therapies to treat hematologic and solid cancers. The company filed an investigational new drug (IND) application with the FDA for its product K-NK002 in April 2020. Kiadis is led by CEO Arthur Lahr, who joined the firm in 2017.
CEO: Bobby Gaspar
Orchard Therapeutics, founded in 2015, has bases in London, San Francisco and Boston. Orchard focuses on treating rare, inherited disorders, particularly in children, utilising advanced gene therapy technology to correct genetic disorders via a single treatment. The company’s main focus areas are neurometabolic disorders, primary immune deficiencies and blood disorders. Orchard is partnering with gene company Généthon to develop an ex vivo autologous gene therapy for X-linked chronic granulomatous disease (X-CGD). The firm also acquired GSK’s gene therapy portfolio for rare inherited diseases, including flagship product Strimvelis, which was the first ex vivo autologous gene therapy to be approved by the European Medicines Agency (EMA). Bobby Gaspar was appointed CEO of Orchard in March 2020, succeeding Mark Rothera.
Euronext Brussels, Paris, Nasdaq: CYAD
CEO: Filippo Petti
Celyad is a biopharmaceutical company that specializes in CAR-T cell therapy and is developing technologies aimed at treating cancer and other diseases with poor prognosis. Celyad boasts two immuno-oncology technological platforms, one autologous and one allogeneic. Its pipeline includes CYAD-01, currently in Phase I trials in patients with Acute Myeloid Leukemia. Its most notable partnership is with Novartis, in which the firm has granted Novartis a non-exclusive license for its allogeneic TCR-deficient CAR-T cells patents. Preliminary findings have shown that the NKG2D receptor in CYAD-01 is able to bind to multiple ligands, meaning that CYAD-01 not only targets and kills the tumour but also its entire micro-environment. Headquartered in New York and in Mont-Saint-Guibert, Belgium, Celyad is directed by CEO Filippo Petti.
CEO: Walter Flamenbaum
AgenTus Therapeutics, founded in 2017, is composed of expert scientists working in offices in Lexington USA, Cambridge UK, and Waterloo Belgium and led by CEO Walter Flamenbaum. AgenTus harnesses its drug discovery platform and other proprietary antigen receptor discovery platforms to identify, characterize and engineer novel Chimeric Antigen Receptors and T Cell Receptor adoptive cell therapy candidates. The firm’s ultimate aim is to develop living drugs to treat a wide range of cancer patients. Its allogeneic format candidate, Native iNKT, is currently in its final preclinical phase and waiting to enter Phase I trials. AgenTus counts on collaborations as a key strategy and currently has partnerships with NYU Langone Health, Masthercell, and Be The Match.
Nasdaq: Cellectis ADS (Nasdaq), Euronext Paris: Cellectis
CEO: André Choulika
Cellectis “is working to develop the concept of off-the-shelf, gene-edited UCART cells – universal CAR-T,” according to an interview with CEO André Choulika. Cellectis aims to leverage UCART to revolutionise cell therapy, touting it as a universal gene-edited solution as opposed to the current therapies on the market which uses a patient’s own re-engineered T-cells. Cellectis uses its flagship technology TALEN® to gene-edit CAR T-cells from healthy donors into “off-the-shelf” immunotherapy product candidates designed to work for a large number of patients. Cellectis has 6 products in the pipeline past IND filing state, including UCART19, an allogeneic CAR T-cell product candidate. Servier has acquired exclusive worldwide rights to further develop and commercialize UCART19, which is now being jointly developed by Servier and Allogene. Cellectis is also bringing to market its wholly controlled product candidate UCART123, which is a gene-edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in acute myeloid leukaemia (AML). Cellectis is based in New York, Paris, and the Research Triangle city of Raleigh, North Carolina. André Choulika is one of the founders of Cellectis and has been its CEO since the company’s founding in 1999.