Rigenerand receives regulatory approval for gene therapy production in Italy
Massimo Dominici, scientific founder, Rigenerand

Rigenerand receives regulatory approval for gene therapy production in Italy

Rigenerand receives regulatory approval
for gene therapy production in Italy

The italian biotech company Rigenerand has received received authorization from the Italian Medicine Authority (AIFA) to produce gene therapy medicinal products for clinical purposes. This approval enables Rigenerand to manufacture its own product, RR001, for the treatment of pancreatic cancer. RR001 is based on autologous human adipose perivascular stromal cells genetically modified to secrete soluble TRAIL (Tumor Necrosis Factor-related Apoptosis-inducing Ligand) protein. Since this protein induces cell death, injection of RR001 into tumors allows production of TRAIL that will bind to its receptors on the surface of cancer cells and trigger their death. RR001 has been classified by European Medicines Agency as gene therapy. It has also received Orphan Drug Status designation in the US in May 2018 and Orphan Medicinal Product designation in Europe in November 2018. « The regulatory approval for Rigenerand to produce gene therapies for clinical development now enables Rigenerand to enter the clinic with its own gene therapy product to target pancreatic cancer, » said Massimo Dominici, scientific founder, Rigenerand. « Combined with the Orphan Drug Designation, the approval will enable Rigenerand to choose an accelerated pathway to bring a gene therapy approach to pancreatic cancer patients with little alternative therapeutic option. » Rigenerand will now be able to initiate the phase I clinical trial of RR001, expected to start in Q1, 2021.

New consultancy services

Founded  as spin-off of the University of Modena and Reggio Emilia, Rigenerand will also now offer direct GMP CDMO services to international and Italian partners in clinical development of cell and gene therapy products. The company plans to expand its manufacturing facility from five sterile clean rooms suits. This will be by implementing further closed system and isolator technology in pre-designated areas in its facilities. The five sterile cleanrooms are within the Rigenerand GMP facility, which contains a Biosafety Level 3 (BSL3) negative pressure area, suitable to handle genetically modified microorganisms, viruses and Risk Group 3 microorganisms, as well as a Biosafety Level 2 (BSL2), positive pressure area: suitable to manipulate non-infectious cell based products and Risk Group 2 microorganisms. In addition, Rigenerand is now authorized to deliver consultancy to biotechnology and pharmaceutical companies on cell and gene therapy development and manufacturing. This consultancy includes expanding process capabilities and developing early-stage cell and gene therapy medicinal products for clinical purposes. « The authorization is also essential in allowing Rigenerand to offer its much needed GMP CDMO services to the wider cell and gene therapy sector, » said Giorgio Mari, Rigenerand CEO. « We will be expanding our CDMO facility to cater for increasing demand. Operating as both a developer with a clinical pipeline as well as a CDMO has resulted in an unrivalled blend of expertise for us to offer to partners and the wider cell and gene therapy industry. »

June 6, 2020