List of Gene Therapy Companies in Italy - 16

Alia Therapeutics is a pioneering gene editing company with a vision to revolutionize medicine by curing genetic diseases at their roots. Alia’s versatile portfolio of novel CRISPR systems expands the range of targetable genomic sites and increases the precision of genetic modifications, bringing gene editing to the next level of specificity and safety, which are required for therapeutic applications.

Altheia Science is a pioneering start-up founded by two world renowned Italian scientists, Prof. Alessandra Biffi, MD and Prof. Paolo Fiorina, MD, PhD, together with AurorA-TT. Altheia Science’s pipeline exploits pioneering therapeutic tools for autoimmune diseases and cancer. The modulation of PD-L1 expression at the molecular and protein level is key to devise advanced treatments. This can be achieved by first-in-class molecules controlling the PD-L1 pathway and/or by lentiviral vector-based engineering of patients’ hematopoietic stem cells. Altheia Science’s approach intends to drastically modify the natural history of diseases with high clinical impact in autoimmunity and cancer by modulating PD-L1 expression, achieving tangible and durable benefit for each patient.

Anemocyte is an Italian Biotech Manufacturing Organization with a strong focus on the development, manufacturing, and analytical services to address needs in the field of: • pDNA – Plasmids manufacturing (R&D, Hiqh-Quality, and GMP) for Viral Vector and mRNA; • mRNA – mRNA manufacturing (R&D) for vaccines and therapeutic treatments. Our experience: • More than 20 years of development and manufacturing experience (from R&D to GMP) in the field of Cell and Gene Therapies and Nucleic Acids. • Over the years, Anemocyte has been involved in multiple projects with a wide range of complexity.

BIOGENERA SpA is a company operating in the biotechnology pharmaceutical sector, specialising in the research and development of new DNA-based patient-specific biotechnological medicines for the treatment of serious pathologies. Founded in 2008, with its headquarters in Bologna, BIOGENERA SpA has a multidisciplinary team of researchers and is supported by numerous University partnerships ( in particular, the University of Bologna) and other companies of the pharmaceutical sector.

Borea Therapeutics develops next-generation gene therapy vectors using a knowledge-based capsid design platform

Cellply is a life-science company focused on developing next-generation analytical tools enabling a deep characterization cancer immunotherapies, cell therapies and cell-based biotherapeutics. Our goal is to support the development of novel cancer immunotherapies by accelerating the R&D, advancing the quality control and monitoring the clinical response to these treatments. Through the use of proprietary microfluidic technologies, highly-automated analytical instruments and AI-powered image and data analytics, the Cellply platform defines the function of the immune system one cell at a time, combining functional data with other multiplex data and providing an unprecedented single-cell analytical solution.

Galileo Research origins lie in 1972 as the Research Centre of Istituto Gentili, an Italian pharmaceutical company founded in 1917. At the beginning of the ‘80s, scientists at the Gentili Research Centre discovered and developed new molecules in the class of bisphosphonates and in the field of bone diseases, in particular clodronate and alendronate. The Research Centre continued to work in the field of bone and joint metabolism and also expanded its knowledge and activities to the areas of oncology and CNS. Galileo Research was established in July 2011.

Genenta (Nasdaq: GNTA) is a clinical stage immuno-oncology company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors. Our platform is not tumor type nor target antigen restricted and provides sustained targeted expression of therapeutic payload(s) inside the tumor micro environment. Genenta’s lead product candidate, Temferon™, precisely targets the delivery of interferon-alpha to the tumor micro-environment, minimizing systemic toxicity while breaking tumor-induced immune tolerance. Our treatments are designed as one-time monotherapies, but with the additional potential, when used in combination, to significantly enhance the efficacy of other approved therapeutics.

Genespire is a biotechnology company focused on the development of durable and transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners.

Heqet Therapeutics is a company that develops groundbreaking genetic medicines to reverse the damage of ischemic heart disease, the leading cause of death worldwide. Their therapeutic approach aims to address the underlying drivers of heart disease.

Holostem Terapie Avanzate is the first biotechnological company entirely devoted to development, manufacture, registration and distribution of Advanced Therapies Medicinal Products (ATMPs) based on cultures of epithelial stem cells both for cell and gene therapy. The main aim of Holostem Terapie Avanzate is to promote epithelial stem cell-based Regenerative Medicine for patients with no alternative therapeutic solutions. Holostem Terapie Avanzate is located into the Centre for Regenerative Medicine "Stefano Ferrari"​ of the University of Modena and Reggio Emilia

The International Centre for Genetic Engineering and Biotechnology ICGEB is an intergovernmental research organisation that operates within the United Nations common system. It was established in 1983 as a Centre for excellence in research, training and technology transfer to industry in the field of biotechnology to promote sustainable global development. With over 60 Member States and 40 Affiliated Centres worldwide, it runs 46 research programmes in its own laboratories in Trieste, Italy, New Delhi, India and Cape Town, South Africa. Almost 700 people work at ICGEB comprising 550 scientific personnel. The organisation has generated almost 3000 scientific publications in the Life Sciences since 1988 and currently holds over 80 active research grants. ICGEB has awarded almost 1400 fellowships, with over 300 fellows and 150 PhD students currently on board. It has organised over 560 Meetings and Courses worldwide and has funded over 550 Collaborative Research Programme-Research Grants. It has produced and distributed over 600 movies (podcasts) of high-scientific content in 50 topics in the Life Sciences - that are freely available for download on iTunes, YouTube and on the ICGEB Web site. Active in Public Engagement and on Social Media networks, ICGEB invites, in particular, its Alumni to join the ICGEB Alumni Group on Linkedin. http://www.icgeb.org

Politecnico Milano is a scientific-technological university which trains engineers, architects and designers. The University has always focused on the quality and innovation of its teaching and research, developing a fruitful relationship with business and productive world by means of experimental research and technological transfer. Research has always been linked to didactics and is a priority commitment which has allowed Politecnico Milano to achieve high quality results at an international level as to join the university to the business world. Research constitutes a parallel path to that formed by cooperation and alliances with the industrial system. Knowing the world in which you are going to work is a vital requirement for training students. By referring back to the needs of the industrial world and public administration, research is facilitated in following new paths and dealing with the need for constant and rapid innovation. The alliance with the industrial world, also favored by Fondazione Politecnico and by consortiums to which Politecnico belongs, allows the university to follow the vocation of the territories in which it operates and to be a stimulus for their development. The challenge which is being met today projects this tradition which is strongly rooted in the territory beyond the borders of the country, in a relationship which is developing first of all at the European level with the objective of contributing to the creation of a single professional training market. Politecnico takes part in several research, sites and training projects collaborating with the most qualified European universities. Politecnico's contribution is increasingly being extended to other countries: from North America to Southeast Asia to Eastern Europe. Today the drive to internationalization sees Politecnico take part in the European and world network of leading technical universities and offers several beside many which are entirely taught in English.

ReiThera is a company devoted to developing and producing biopharmaceutical products based on gene delivery technologies doe advanced therapies, in order to prevent and treat several serious or life-threatening diseases.