Evaluating the attractiveness & potential positioning of a new kinase inhibitor

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...
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Gene therapy technology opportunity assessment

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...
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Indication triaging for AAV-based gene therapies

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...
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AAV gene therapy roadmap to IND, for a cardiac indication

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...
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Gap analysis & pre-IND meeting preparation for preclinical compound

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...
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CMC and regulatory affairs gene therapy support

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...
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Due Diligence of Late Stage Gene Therapy Asset

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...
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Pipeline strategy for gene therapy company

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...
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Commercial Development plan for beta thalassemia gene therapy

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...
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Gene therapy manufacturing license negotiation

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...
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Developing a Target Product Profile (TPP) for a novel gene therapy

Challenge A privately-held biotechnology company developing and exploiting a proprietary technology platform for optimizing and controlling gene therapy expression was looking to evolve its business...
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Assessment of an AAV gene therapy for an ophthalmic indication

Challenge: Alacrita conducted an assessment of an AAV gene therapy for an ophthalmic indication, focusing on market potential, technical and regulatory issues and identification of key risks.
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