Rigenerand receives regulatory approval for gene therapy production

Authorisation has enabled Rigenerand to enter into clinic, offer CDMO services and consultancy, and plan to expand cleanroom capacity

Rigenerand, a biotech company that both develops and manufactures medicinal products for cell therapy applications, primarily for regenerative medicine and oncology, has announces it has received authorisation from the Italian Medicine Authority (AIFA) to produce gene therapy medicinal products for clinical purposes.

This authorisation enables Rigenerand to manufacturer its own autologous gene therapy medicinal product (RR001) for the treatment of pancreatic cancer. It also authorises the company to start its phase I first-in-man clinical trial of RR001, now expected to start in Q1, 2021.

RR001 has been granted Orphan Drug Designation (ODD) by both the US FDA and European Commission COMP (Committee for Orphan Medicinal Products). This ODD offers the opportunity for Rigenerand to request an accelerated assessment procedure and quicker development pathways towards a marketing authorisation in the US and EU.

Rigenerand will also now offer direct GMP CDMO services to international and Italian partners in clinical development of cell and gene therapy products (ATMPs). The company will utilise its experience from its drug development and diagnostic arms to deliver a science-based approach to its GMP manufacturing services and cell-based medicinal products development.

Rigenerand plans to expand its manufacturing facility from five sterile cleanrooms 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 (MOGM), 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. Rigenerand's cleanroom technology offers the flexibility to scale-up the processes from academic / hospital laboratories, and the feasibility of technology transfer of manufacturing processes from other cell factories in order to expand their process capability.

In addition, Rigenerand is now authorised 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.

As the cell and gene therapy sector continues to grow, with increased numbers of therapies moving through clinical development and onto commercialisation, demand for CDMO services will continue to grow. There is an increased demand for global networks of CDMO GMP cell and gene therapy manufacturing. This calls for an improved capacity to treat patients whilst reducing logistical complexities, issues, risks, and costs.

"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."

"The authorisation 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."