4:14 PM
Apr 11, 2019
 |  BC Innovations  |  Targets & Mechanisms

A venture view of biological white space

VCs on BioCentury’s SAB weigh in on biological whitespace

Scanning the horizon of translational research, VCs on BioCentury’s scientific advisory board see innate neuroinflammation as a white space area flush with under-tapped angles for drug development, and think the rise of single cell-level data will be a wellspring of selectively targeted medicines across many indications.

In a roundtable discussion with BioCentury, 5 AM Ventures Managing Partner Kush Parmar, Flagship Pioneering Venture Partner Paul-Peter Tak and Third Rock Ventures Partner Reid Huber identified targets and tools that could form the basis for new classes of therapies.

Their perspectives point to a near-term future that will see microglia and stromal cells, long dismissed as supporting actors, take center stage as key modulators of disease.

Single cell technologies, once the provenance of individual groups and centers, are going mainstream through international consortia developing shared datasets and analytical tools. The conversation is no longer about whether the methods can deliver actionable insights, but about how to execute on them via multi-targeting therapeutic modalities.

And having all but overtaken oncology, immunology is now poised to have a major impact on neurology, where the cross-talk with innate immunity is yielding new therapeutic strategies for neurodegenerative and autoimmune diseases.

Some areas called out by the VCs, like microglial biology and inflammasome activity in the brain, are already the focus of a handful of company programs that have entered the clinic or been snapped up through M&A.

In other newer areas, like drugging higher order genome structure, companies are finding ways to test the waters via traditional strategies such as targeting protein regulators or conducting phenotypic screens.

Expanding neuroinflammation

Neuroinflammation has already become a core pillar of work in neurodegenerative disorders, particularly Alzheimer’s disease (AD), where companies are increasingly looking for alternatives to the well-trodden territory of β amyloid aggregation and cholinergic signaling (see “After Amyloid”).

But the biology of microglia -- the CNS’s primary immune cells -- has been poorly understood, according to Third Rock’s Huber. “It’s been a very difficult area of biology to explore. The translational models have lagged, to say the least.”

Advances in more relevant model systems, including human induced pluripotent stem (iPS) cells and organoids, are opening up new avenues of research, said Huber.

A spate of studies in 2017 described increasingly efficient methods for generating iPS cell-derived microglia, and a...

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