evotec_Nov23_PanOmicsDriven - 7

" To improve outcomes, we need to improve
disease management, which is powered by improved
objective measurement, "
William Hagstrom, the founder and CEO of Octave Bioscience
TARGETING THE KINASE TREE
More than a decade ago, Blueprint Medicines
was launched as a developer of personalized medicines.
Since then, Blueprint has centered its discovery
efforts around the 518 kinases encoded by the
human kinome.
For most of these kinases, functions remain
unclear. However, by studying these kinases, the
company expects to identify kinase variants that
drive the growth of varied cancers.
" Kinase medicines are designed to block the
activity of protein kinases-enzymes that regulate
critical biochemical pathways in cell growth,
survival, metabolism, and immune activity, "
explains Kate Haviland, Blueprint's president and
CEO. " However, early-generation kinase medicines
were limited by poor selectivity, off-target effects,
and the emergence of resistance.
" The challenge, historically, has been that
toxicity is driven by both activity against the wildtype
protein as well as off-target activity on other
tyrosine kinases. But we've been able to make molecules
that are highly selective and combinable, and I
Blueprint has two commercially approved
products. The first product is avapritinib. (In the
United States, the trade name is Ayvakit; in Europe,
it is Ayvakyt.) Avapritinib gained FDA approval
for patients with advanced systemic mastocytosis
in June 2021, and it gained European Commission
approval in April 2022. The product is also approved
for a genomically defined subset of patients with
gastrointestinal stromal tumors. The second product
is pralsetinib (trade name: Gavreto). Pralsetinib
is approved for patients who have metastatic or
advanced RET-altered cancers, such as non-small
cell lung cancer or medullary thyroid cancer.
think this is really the beginning of a new journey. "
Haviland indicates that Blueprint has developed
a proprietary scientific platform that allows the
company to rapidly and reproducibly design medicines
that selectively target kinase drivers of disease,
enabling improved potency, less off-target activity,
and an increased probability of clinical success.
She remarks, " We believe we are the only biotechnology
company to discover two novel programs
and advance them both into the clinic and through
multiple regulatory approvals in less than 10 years. "
7
GENengnews.com
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evotec_Nov23_PanOmicsDriven

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