Produits de thérapie génique
Collategene
ANGES
A gene therapy consisting of an intramuscular injection of plasmid (ADN) codant pour le facteur de croissance des hépatocytes (HGF), destiné à développer des vaisseaux sanguins, pour le traitement de l'ischémie critique des membres. Approved in Japan in February 2019.

Gendicine
SHENZHEN SIBIONO GENETECH
A recombinant adenovirus engineered to express wildtype-p53 (rAd-p53), designed to treat patients with tumors which have mutated p53 genes. Gendicine is the first gene therapy product approved for clinical use in humans, approved in China in 2003.

Adcétris
AMGEN
IMLYGIC is a weakened form of Herpes Simplex Virus Type 1, which is commonly called the cold sore virus, indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery. Approved by EMA in December 2015, US FDA in October 2015, Australian TGA in June 2016.

Libmeldy
ORCHARD THERAPEUTICS
A lentiviral vector-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). Approved by the European Commission in December 2020.

Luxturna
SPARK THERAPEUTICS
LUXTURNA is an adeno-associated viral vector gene therapy indicated for the treatment of RPE65-mediated inherited retinal dystrophies. Approved by U.S. FDA in December 2017; approved by the EMA in November 2018; approved by Health Canada in October 2020.

Roctavian
BIOMARIN
Roctavian is a gene therapy for the treatment of severe hemophilia A in adult patients. It was granted conditional marketing authorization in the European Union in August 2022 to treat Hemophilia A in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Skysona
BLUEBIRD BIO
Skysona is gene therapy made using a genetically modified autologous CD34+ cell-enriched population that contains hematopoietic stem cells. It was approved by the EMA in 2021, but was later withdrawn from the region by the developer. It was approved by the US FDA in 2022 to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).

Strimvelis
GSK
Strimvelis is an ex-vivo stem cell gene therapy which uses retroviral vector encoding adenosine deaminase gene transfer into hematopoietic stem/progenitor cells. Strimvelis is indicated for the treatment of adenosine deaminase severe combined immune deficiency. Approved by the EMA in May 2016.

Upstaza
PTC THERAPEUTICS
Upstaza is a gene therapy medicine that is used in adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency. It was approved by the European Commission in July 2022 to use in patients 18 months and older with a clinical, moléculaire, and genetically confirmed diagnosis of aromatic L‑amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Zolgensma
NOVARTIS GENE THERAPIES
Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy. Zolgensma was approved by the FDA for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA), including those who are pre-symptomatic at diagnosis, en mai 2019. The therapy was approved in Japan (Mars 2020), the European Union for certain forms of SMA (Peut 2020), Éviter les systèmes de défense de l'hôte (décembre 2020), Australie (Mars 2021), and South Korea (Peut 2021).

Zynteglo
BLUEBIRD BIO
Zynteglo is an ex-vivo lentiviral based gene therapy used to provide a functional copy of the the β-globin gene into a patient’s hematopoietic (du sang) cellules souches. Received EU marketing authorization in June 2019 and was approved by the US FDA in September of 2022.


NBScience

organisme de recherche sous contrat

thérapie par cellules souches