RNA - Genetherapys
Who we are


A Scientific Institute for Research, Hospitalization and Health Care and an Academic Medical Center. OPBG is Italy’s main pediatric hospital and acts as a referral center at national and international level. The hospital stands out for its pioneering and innovative role in advanced cell and gene therapies. Indeed, OPBG scientists have a relevant record of accomplishment in research and clinical experience within the field of Gene and Cell Therapy (GCT). The academic OPBG GMP Facility, approved by the national competent authority (AIFA) for GCT, has relevant experience in the transduction of somatic human cells with viral vectors (retro and lentiviruses). In the contest of the Natioanl Center, OPBG will be the coordinator of Spoke 10.

Mattia Algeri
Medical Director

Settore studi clinici oncoematologici e terapie cellulari

Biagio De Angelis
Project Manager

Unità terapia genica dei tumori

Francesca Del Bufalo
Medical Director

Settore studi clinici oncoematologici e terapie cellulari

Monica Gunetti
Qualified Person - Pharmaceutical Manager

Officina Farmaceutica

Stefano Iacovelli
Production Manager

Settore produzione Officina Farmaceutica

Franco Locatelli
Lead Manager, Clinical Project Manager

Settore studi clinici oncoematologici e terapie cellulari

Pietro Merli
Medical Director

Settore studi clinici oncoematologici e terapie cellulari

Daria Pagliara
Medical Director

Settore studi clinici oncoematologici e terapie cellulari

Concetta Quintarelli
Senior Researcher - Manager

Unità di terapia genica dei tumori

Mara Vinci
Senior Researcher

Unità di genetica ed epigenetica dei tumori pediatrici

Marco Berettoni
Senior GMP Operator

Settore assicurazione qualità Officina Farmaceutica

Gaetano D'amore
Intermediate GMP Operator

Settore assicurazione qualità Officina Farmaceutica

Stefano Di Cecca
Senior Technologist

Unità di terapia genica dei tumori

Clelia Geraci
Junior Technologist

Unità di terapia genica dei tumori

Giulia Grisanti
Intermediate GMP Operator

Settore produzione Officina Farmaceutica

Laura Iaffaldano
Senior Researcher

Unità di terapia genica dei tumori

Simona Manni
Senior Researcher

Unità di terapia genica dei tumori

Marilena Margiotta
Expert GMP Senior Biologist

Unità di terapia genica dei tumori

Natascia Perciballi
Intermediate GMP Operator

Settore controllo qualità Officina Farmaceutica

Eleonora Scarano
Intermediate GMP Operator

Settore assicurazione qualità Officina Farmaceutica

Flavia Trionfetti
Junior GMP Operator

Settore produzione Officina Farmaceutica

Adriana Valisareanu
GMP Sanitation Operator

Settore produzione Officina Farmaceutica

Michele Veneruso
Expert GMP Junior Biologist

Unità di terapia genica dei tumori


Tettamanti Foundation has consolidated expertise in the preclinical characterization of CAR-T cells against ALL and AML developing in recent years a non-viral gene transfer approach that has been patented (EP3215605: Improved method for the generation of genetically modified cells) for the production of CAR-CIK cells.

Giuseppe Gaipa
Biology Director

Giovanna D’Amico
Biology Director

Marta Serafini
Head of Research

Alice Silvia Pievani
Biology Researcher

Sarah Tettamanti
Biology Researcher

Cristina Bugarin
Assistant Biologist

Grazia Fazio
Biology Researcher

Erica Dander
Biology Researcher


San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) aims to advance biomedical research toward the diagnosis, cure, and prevention of human rare genetic diseases. FT counts two intramural research Institutes: the Telethon Institute for Genetics and Medicine (TIGEM) located in Pozzuoli and the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) located in Milan. SR-TIGET has provided seminal contributions to the gene and cell therapy field with relevant discoveries in vector design, gene transfer, and gene editing strategies, which have led to several successful trials of novel ATMPs with >140 patients treated and 2 products already approved on the EU market. In addition, SR-TIGET comprises expertise and facilities for ATMP process development and scale-up as well as a GLP Assays Center certified by ISS for method validation and in vivo biodistribution/toxicology studies of ATMP.

Claudia Forni
Translational Project Manager

Martina Ragazzi
Translational Project Manager

Marina Radrizzani
Senior Researcher

Stefano Zancan
Head of Clinical Development and Operations

Paola Albertini
Senior Researcher

Carolina Cuofano
Junior Technologist

Federica Fardella
Post-Doc Researcher

Beatrice Martini
Junior Technologist

Sara Rinaldi
Junior Technologist

Evelyn Oliva Savoia
Junior Researcher

Maria Sole Vespasiano
Junior Researcher


National Research Council of Italy (CNR) scientists have provided significant contributions to several research activities with high scientific relevance with an understanding of the impact of RNA metabolism on human health. CNR explores novel delivery methods for therapeutic molecules, including the development of new AAV vectors for the therapy of neurological diseases.

Giuseppe Gigli
Research Manager

Ilaria Elena Palamà
Researcher

Loretta Laureana Del Mercato
Researcher

Stefano Leporatti
Research Manager

Francesca Gervaso
Researcher

Eloisa Sardella
Researcher

Roberto Gristina
Researcher

Alessandro Polini
Researcher

Elisa De Luca
Researcher

Pamela Mozetic
Researcher

Bruna My
Phd student

Ludovica Rizzo
Phd student

Claudia Leone
Phd student

Simone Schirinzi
Phd student


University of Campania (UNICAMPANIA) participants have consolidated expertise in defining gene therapy for inherited retinal dystrophies (IRDs) confirmed by the participation to the first ocular gene therapy clinical trial worldwide for the treatment of an IRD (i.e. Leber Congenital Amaurosis caused by mutations in the RPE65 gene).

Francesca Simonelli
Full professor

Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche

Paolo Melillo
Associate professor

Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche

Settimio Rossi
Associate professor

Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche

Francesco Testa
Associate professor

Dipartimento Multidisciplinare di Specialità Medico-Chirurgiche e Odontoiatriche

Aniello Russo
Full professor

Dipartimento di Scienze e Tecnologie Ambientali Biologiche e Farmaceutiche (DISTABiF)

Sandro Banfi
Full professor

Dipartimento di Medicina di Precisione

Maria Rosaria Barillari
Associate professor

Dipartimento di Salute Mentale e Fisica e Medicina Preventiva

Stefania Mele
Researcher RTD-A

Dipartimento di Economia

Girolamo Di Maio
Researcher RTD-A

Dipartimento di Medicina Sperimentale


University of Padua (UNIPD) has large experience with GMP grade production of vectors for gene therapy, testified by successful clinical trials and by their past direction of the Gene Therapy Program, Boston Children’s Hospital, and their launch of dedicated startups like Altheia Science.

Alessandra Biffi
Full professor

Dipartimento di Salute della Donna e del Bambino

Barbara Buldini
Associate professor

Dipartimento di Salute della Donna e del Bambino

Raffaella Colombatti
Associate professor

Dipartimento di Salute della Donna e del Bambino

Lara Mussolin
Associate professor

Dipartimento di Salute della Donna e del Bambino

Luca Persano
Researcher RTD-B

Dipartimento di Salute della Donna e del Bambino

Martina Pigazzi
Associate professor

Dipartimento di Salute della Donna e del Bambino

Francesco Piazza
Associate professor

Dipartimento di Medicina

Giampietro Viola
Associate professor

Dipartimento di Salute della Donna e del Bambino

Claudia Tregnago
Researcher RTD-A

Dipartimento di Salute della Donna e del Bambino

Raffaele Mattera
Phd student

Dipartimento di Salute della Donna e del Bambino

Silvia Merlini
Phd student

Dipartimento di Salute della Donna e del Bambino


Gabriele d’Annunzio University of Chieti and Pescara (UNICH) scientists have strong scientific experience on several relevant scientific topics likewise leukemia, as well as on the study of T cells and extracellular vesicles. The scientist team operates at the Center for Advanced Studies and Technology (CAST), where they conduct synergistic activities cooperating with the core facilities (genomic, proteomic, microscopy, flow cytometry) to develop and translate several cell therapies to clinical practice.

Alessandro Cama
Full professor

Dipartimento di Farmacia

Mauro Di Ianni
Associate professor

Dipartimento di Medicina e Scienze dell’Invecchiamento

Natalia Di Pietro
Researcher RTD-A

Dipartimento di Scienze Mediche, Orali e Biotecnologiche

Manuela Iezzi
Associate professor

Dipartimento di Neuroscienze, Imaging e Scienze Cliniche

Paola Lanuti
Associate professor

Dipartimento di Medicina e Scienze dell’Invecchiamento

Lisa Marinelli
Researcher RTD-A

Dipartimento di Farmacia

Assunta Pandolfi
Full professor

Dipartimento di Scienze Mediche, Orali e Biotecnologiche

Nicola Tinari
Associate professor

Dipartimento di Scienze Mediche, Orali e Biotecnologiche

Serena Veschi
Researcher RTD-A

Dipartimento di Farmacia

Rosa Visone
Associate professor

Dipartimento di Scienze Mediche, Orali e Biotecnologiche

Rosalba Florio
Researcher RTD-A

Dipartimento di Farmacia

Sara Pagotto
Researcher RTD-A

Dipartimento di Scienze Mediche, Orali e Biotecnologiche

Pasquale Simeone
Researcher RTD-A

Dipartimento di Medicina e Scienze dell’Invecchiamento

Davide Brocco
Researcher RTD-A

Dipartimento di Scienze Mediche, Orali e Biotecnologiche


Alessandro Aiuti
Full professor

Maria Ester Bernardo
Associate professor

Alessio Cantore
Researcher RTD-B

Giuliana Ferrari
Full professor

Angelo Leone Lombardo
Associate professor

Luigi Naldini
Full professor

Renato Ostuni
Associate professor


Takis Biotech provides significant contributions to the discovery and development of innovative Vaccines based on viral vectors and DNA/mRNA platform technology, leveraging on its strong record of accomplishment in drug discovery. As one of its main assets, in vivo electro-gene-transfer (EGT) can be used for a variety of clinically useful applications, from vaccine development to somatic gene therapy.

Erika Salvatori
Researcher

Lucia Lione
Researcher

Grazia Vitagliano
Researcher


IRBM Research Group is an organization with world-class expertise and capabilities in the discovery and development of peptide, small molecule, and antibody-based therapeutics and a strong track record of developing drugs that have entered the different phases of clinical development and in some cases have been approved for marketing. In collaboration with other members of the spoke, IRBM will use its proprietary high-complexity monoclonal antibodies phage library to screen biospecimens from patients with hematologic malignancies to identify novel targets and novel antibodies, which could be used for CAR-T or NK-CAR cell therapy.

Alessandro Carugo
Head of Unit

Andrea Cacciotti
Researcher

Ramona Sterpa
Researcher

Nicole Zampetti
Researcher


InnovaVector is an innovative start-up born as NewCo of the Telethon Foundation, absorbing the skills of the Adeno-Associated Viral (AAV) Vector Core group of the Telethon Institute of Genetics and Medicine (TIGEM). InnovaVector’s team boasts consolidated experience in the production of AAV vectors from R&D to GMP grade, to support the pre-clinical and clinical development of AAV-based products.

Miriam Centrulo
Development and technology transfer specialist

Monica Doria
Research grade Production Manager

Sara Fabbricino
Analytical Development Specialist

Valentina Giuliano
Quality Control Specialist

Carolina Iodice
Research grade Production Senior Specialist

Eleonora Leggiero
Development and tech transfer senior specialist

Bernadette Monforte
Research grade Production Specialist

Christel Gael Ouengue Mbele
Head of Development and technology transfer unit

Fabio Perini
Technical and Project Manager

Chiara Piscopo
Quality Control Manager

Viola Schiano Di Cola
Quality Control Specialist


Performing Beyond Limits (PBL Srl) is an innovative SME whose core business is the design and realization of customized automated industrial lines for biomedical and pharmaceutical sectors. A part from high expertise in the development of inspection and formulation industrial systems, PBL has gained a well-proven experience and know-how in the development of innovative platforms for ATMPs (e.g.. cell and gene therapy, tissue engineering, RNA-based vaccines…). PBL represents a key partner for creation of customized and flexible automated machines for the manufacturing of RNA&Gene therapies, making them more affordable, scaled-up and standardized.

Federica Sartori
Research and Development

Lucian Radu Olteanu
Project Manager

Luca Gagliardi
Automation Manager

Giacomo Zermani
Electrical Engineering and Software Manager

Mario Meduri
Mechanical Designer

Filippo Begarani
Product Innovation Manager

Emanuela Setti
Quality Health and Safety

Alessandro Maffei
Automation Programmer

Beatrice Balboni
Research and Development

Mattia Corrias
Mechanical Designer


Mariangela Amoroso
Country Medical Lead & Medical Head Specialty Care

Mattia Battistini
Healthcare Solution & Innovation Manager