Special Purpose Exit Company (SPEC)
A Special Purpose Exit Company (SPEC) is built around a Rare Disease. Its sole purpose is to build an attractive set of drug assets for adoption by big Pharma. By containing all the necessary documentation on the drugs within a company, the portability is high and the assets becomes more attractive. In forming a SPEC, we partner with Patient Advocacy Groups (PAGs), researchers, clinicians, and biotech entrepreneurs. They help us develop the population database, biomarker tools, and drug modalities that will be documented and owned within the SPEC.
SPEC Ownership
PAGs, along with others, have vested ownership. A key value addition that is unique to a PAG is their patient families. PAGs can nurture the growth of patient databases and make sure that relevant data for trial endpoints is being collected. Devinebio brings a depth of knowledge and expertise in drug discovery and development via its extensive network of collaborators. A Coordinator from Devinebio acts as “CEO” to oversee and make sure the asset development is proceeding on schedule. Finally, investment by Angels, VCs, philanthropy, or other groups, helps finance the activities needed to progress a genetic medicine from target to clinical efficacy.
SPEC Drug Assets
Drug Modality Types
The fastest route to establishing a therapy pipeline is drug repurposing. There is a good chance that an established drug already sold on the market can be repurposed for a rare genetic disease. Other more promising, but more expensive, technologies are ASO, AAV and HSCT. These new methods allow for highly-specific targeting against a genetic deficiency.
ASO stands for Anti-Sense Oligonucleotide. An ASO is a little piece of DNA or RNA that can change gene expression. With antisense DNA, the binding to mRNA triggers degradation of the mRNA message via RNase H activity. With antisense RNA, the sequences are complementary to a target mRNA. Binding of Antisense RNA forms double-stranded regions that block either protein translation or splicing of introns. Both of these ASO types can get rid of proteins that are acting defectively.
AAV stands for Adeno-Associated Virus. AAVs are used as vehicles (or vectors) for highly-targeted gene replacement therapy. They bring into the cell the coding sequence for a missing protein. When properly designed, protein expression is restored to normal levels and the genetic deficiency is corrected for normal gene function.
LV-HSCT stand for Lentivector Hematopoietic Stem Cell Transplantation. This form of gene therapy edits the genome of stem cells to produce a working copy of the missing or altered protein. For some metabolic disorders, this therapy uses the phenomenon of cross-correction, where a patient’s HSCs are modified to express the missing protein and then reintroduced back into the patient so that a populations of modified HSCs can engraft and differentiate into cells that secretion the missing protein thought the body. The secreted protein is taken up by neighboring cells and and normal gene function is restored in the patient.
It depends a lot on the specific gene-associated disease, but often we recommend starting with repurposing to “cut the fastest path” to IND, and then back fill with novel chemicals (NCEs or ASOs) or biologics (AAVs, and LV-HSCTs) to get the best therapy to the patient in the fastest time possible.
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