Defined Health, with more than 30 years of experience, is the leader in the identification and evaluation of new business opportunities for healthcare licensing and business development executives.  We have a unique depth and breadth of understanding of therapeutic categories, built from conducting thousands of opportunity assessments within those categories throughout our history.

Within the regenerative medicines category, Defined Health is engaged by academic groups, non-profit organizations and biopharma firms to help in the early development of gene and cell therapies, among other therapeutic platforms. We are supporting many clients in their efforts to prioritize investment in certain diseases, establishing strategies for developing the evidence for therapeutic value and establishing new business models for healthcare delivery.

Defined Health’s experience in Gene & Cell Therapy includes:

·       Profiling the landscape of numerous rare cancers and non-malignant genetic disease populations across therapeutic categories (hematology, metabolism, endocrinology, neurology, musculoskeletal, etc.)

·        Providing diligence on platform technologies: novel peptide and protein augmentation, substrate reduction, chaperones, gene transfer, cell replacement, CRISPR/Cas9 gene editing, antisense, exon skipping, nonsense read-through, RNAi, miRNA and mRNA loading, across a multitude of rare disorders

·        Assessing therapeutic angiogenic cell and gene therapies for ischemic diseases, myocardial infarction, peripheral artery disease, stroke and regenerative approaches to cartilage and bone disorders

·        Evaluating novel Treg approaches to inducing immune tolerance in autoimmune and inflammatory diseases

·        Thought leadership on prioritizing antigens for adoptive cellular therapies for oncology (CAR T-Cells, TCRs, TILs, CTLs…)

·        Diligence on allogeneic HSC and cord blood ex vivo expansion technology for allogeneic SCT and GvHD

·        Presentations, moderator and panelist participation at symposia and webinars on topics related to gene/cell therapy, rare diseases and Immuno-Oncology

·        Development of a proprietary genetic disease database to expedite the assessment and prioritization of genetic disorders as therapeutic opportunities

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