A UK-based gene therapy company, Freeline Therapeutics, has received a third investment from the UCL Technology Fund, which supports research from University College London. The fund is managed by Albion Ventures in collaboration with UCL Business, the university’s technology transfer office.
Italy-based MolMed SpA has become the seventh company to get a positive recommendation from the European regulator to market an advanced therapy medicinal product, a group that includes cell and gene therapies as well as tissue-engineered products.
As gene editing technologies gain traction in laboratories around the world, companies producing the new technologies are attracting more money from investors. An example is CRISPR Therapeutics which has a number of preclinical projects where it is investigating gene editing for therapeutic use.
The European Medicines Agency has agreed to give an accelerated review of a gene therapy being developed by Kite Pharma Inc that promises to improve the standard of care for patients with diffuse large B-cell lymphoma (DLBCL), a type of blood cancer.
A Phase 1 study of 18 patients with severe osteoarthritis of the knee has shown that a single injection of stem cells derived from the patients’ own adipose tissue was safe and appeared to offer relief from pain.
A startup company founded by Oxford University professors is making waves in the stem cell community because it has technology that promises to control in vivo the destination of cells that can repair and replace tissues affected by disease. The technology will be used to create small molecule drugs that can activate repair mechanisms that already exist in the body to treat major age-related diseases such as cancer, neurodegeneration, heart failure and macular degeneration.
Japanese scientists have reported growing three-dimensional skin tissue in the laboratory and successfully transplanting it into mice where it connected with nerve and muscle tissue allowing the skin to function normally.
Gene and cell therapy executives, meeting in Stockholm in early April, spoke positively about the sector in the wake of the European Medicines Agency’s positive opinion for Strimvelis, a gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an ultra-rare disease.
A new gene therapy developed by the San Raffaele Telethon Institute for Gene Therapy together with GlaxoSmithKline Plc, has been given a positive review by the European Medicines Agency. If approved by the European Commission, it will only be the second gene-based product to reach the market in the west.
Milan-based Genenta Science, which has an experimental gene therapy for delivering interferon to tumours, has signed a multi-year manufacturing agreement with MolMed SpA in preparation for the start of clinical trials of a gene therapy for multiple myeloma.