Commercially, gene therapy is still at the starting line. There are a few approved gene therapy products, but a range of new drugs are expected to be approved by the relevant authorities in the coming years. As research and development continue, so do intensive efforts to resolve issues concerning remuneration systems and regulatory frameworks.
Belief in gene therapy is at a very high level. This is exemplified by, among other things, the large number of clinical studies that are now under way throughout the world. There are currently 376 clinical studies being conducted in the gene therapy field, of which 101 are phase-I studies, 226 are phase-II studies and 49 are phase-III studies (Alliance for Regenerative Medicine, H1 report 2021).
Confidence in gene therapy is also demonstrated by the massive flow of capital into this field. During the first six months of 2021, investment in gene-therapy-related research and development amounted to USD 6,4 billion (Alliance for Regenerative Medicine, H1 report 2021). The emphasis is in the area of oncology, but cardiovascular diseases and diseases related to the central nervous system are also very common.
On August 30, 2017, the US Food and Drug Administration (FDA) approved the first gene therapy for the US market. The approved therapy, Kymriah, from the pharmaceutical company Novartis, has been developed for treatment of pediatric patients and young people with acute lymphoblastic leukemia. Since then, a number of gene therapies have been approved in both the US and Europe, including Luxturna from CombiGene’s partner Spark Therapeutics.
Epilepsy is a global problem. The disease affects an estimated 0.6 to 0.8 percent of the world’s population. In 2016 there were 5.7 million diagnosed epilepsy patients in the USA, the EU4*, the UK, and Japan. About one-third of these patients do not respond to conventional medical treatment. It is this patient group CombiGene intends to help with its candidate drug, CG01.
* EU4 = France, Germany, Italy, and Spain
In the USA alone some 14,000 patients are diagnosed with drug-resistant focal epilepsy each year, patients who could be candidates for surgery. CombiGene estimates that, realistically, 10–20 percent of these patients could be treated with the candidate drug CG01.
CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282
Partial lipodystrophy is a very rare disorder. Estimates place the number of patients in the USA and Europe at about 2,000. Therefore, according to CombiGene’s assessment, there are good prospects for obtaining orphan drug designation for the lipodystrophy project’s candidate drug. If orphan drug designation is granted for the candidate drug, CombiGene will have several significant advantages in the form of regulatory advice, tax relief, public funding, reduced fees and longer market exclusivity.
Given the advantages of an orphan drug designation, CombiGene may potentially develop this project all the way to market under its own management.