5:28 PM
May 23, 2019
 |  BC Innovations  |  Emerging Company Profile

Anima opens new target space in translational control

How Anima is leveraging its translational control platform to pursue new targets, small molecules and big pharma deals

Anima Biotech’s answer to undruggable targets is to go after the machinery that turns their mRNA into protein, adding a new approach to the gene regulation tool kit. The strategy should give Anima a leg up over competitors looking to bind RNA directly, in terms of target stability, tissue selectivity and the ability to tune protein expression up or down.

Anima Biotech Inc. was launched in 2014 with technology from the University of Pennsylvania that lets it visualize translation of almost any protein, enabling it to track how different molecules impact the process.

The platform has spawned a growing pipeline of preclinical translation modulators against novel, undisclosed targets, a $5 million series A round and $25 million series B round from undisclosed private investors, and a 2018 deal with Eli Lilly and Co. that could bring in over $1 billion. Under the Lilly deal, Anima received $30 million up front and $14 million in research funding to identify translation inhibitors of undisclosed targets, and is eligible to receive up to $1.05 billion in milestones, plus royalties.

While Anima is well-funded to advance its own pipeline internally, it is also lining up new deals. The biotech is in discussions with other partners about structuring the same kinds of collaborations that it has with Lilly, and hopes to disclose another deal in the coming months.

Approaches to control gene expression are gaining in popularity as companies push up against the limitations of small molecules and antibodies.

Small molecules can penetrate the cell but require targets with well-defined binding pockets, and antibodies are generally too large to get inside cells. That leaves a vast set of proteins that can’t be drugged with these standard modalities, including the notoriously difficult p53, KRAS and MYC.

Interfering with gene expression offers a workaround by stepping in before the protein is made to either repress or ramp up its expression. The only problem is that most gene control therapies rely on alternative modalities, such as mRNA, antisense oligonucleotides or gene editing tools, all of which come with delivery challenges.

One of the first small molecule...

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