Gene Therapy

Challenge: A biotech company developing novel gene therapy approaches for in vivo cell trans-differentiation required an AAV gene therapy expert to serve on a scientific advisory board (SAB) tasked...

Challenge: A national biomedical research agency requested Alacrita’s support with (1) a technical assessment of the agency’s own internal capabilities in AAV manufacturing, (2) a global landscape of...

Challenge: An in-silico drug discovery company requested an expert review of the robustness and potential positioning of a new kinase inhibitor in oncology and inflammatory disease. Alacrita was...

Challenge: A cell and gene therapy platform company developing a scalable AAV production system, requested Alacrita’s guidance on the priority order for AAV vector capsid serotype release and the...

Challenge: A biopharmaceutical company developing novel immunomodulatory technology was exploring the potential clinical use of its lead candidate in combination with AAV based gene therapies to...

Challenge An academic group was interested in developing and commercializing an AAV vector-delivered gene therapy for a cardiac indication through the formation of a NewCo. They required assistance...

Challenge: Our client planning to develop and commercialize a preclinical compound for metabolic disorders. The product had a wide therapeutic potential for disorders associated with disruption of...

Challenge: A biotech company developing novel gene therapy approaches for in vivo cell trans-differentiation had lead programs in IND-enabling studies for two indications. The client requested...

Challenge: Our client, a global pharmaceutical company, was facing a short deadline to making an offer for a late stage gene therapy asset and was under considerable competitive deal pressure. This...

Challenge: A leading biotechnology company with a focus on development and commercialization of gene therapies for bleeding and other debilitating disorders had developed a next-generation AAV gene...

Challenge: A leading European university identified a gene therapy for the treatment of haemoglobinopathies. Alacrita was asked to support the Principal Investigator (PI) in developing a Commercial...

Challenge A private biotech company developing gene therapy products for several orphan diseases was in the process of initiating CMC development for their pipeline products. In order to move...