A Philadelphia gene-therapy biotech venture just raised $88 million in investment from Johnson & Johnson and other investors, domestic and from Europe, to help accelerate the development of RNA-based drugs to market. Now Aro Biotherapeutics, founded by two former Johnson & Johnson executives, can double the team size. Susan Dillon, the firm’s chief executive officer and co-funder reports they will go from 20 to over 40 scientists and managers over the year with an aim of bringing treatments to clinical trials. Based on Centyrins, that is human proteins engineered in the company’s labs with an aim of targeting hereditary and immune-system diseases.
The Gene Therapy Capital: Philly
Philadelphia appears to be the epicenter of gene based therapy development. With the Spark Therapeutics acquisition (Roche)as well as many other VC funding and IPOs over the past couple years, life science investment capital floods into the City of Brotherly Love.
According to the biotech startup’s founders, both Ms. Dillon and Karyn O’Neil, this approach, that is the use of Centyrins, represents a more optimized for mass production compared to the more “custom-made treatments” other gene-therapy biotech firms are focused on.
What are they?
According to the firm’s website these small, engineered proteins are derived from a human protein known as Tenascin C, found in the extracellular matrix of various tissues. Apparently, endowed with exceptional stability properties, this represents an important consideration for the delivery of therapeutics within the intracellular environment.
This startup has developed libraries containing trillions of Centyrin variants; they can leverage this growing repository to rapidly identify Centyrin clones that bind with high specificity and affinity against antigens of interest.
Moreover, the Aro team have mapped a Centyrin framework that helps identify specific amino acid residues where they can chemically conjugate drug payloads, without impacting underlying stability elements, solubility or antigen binding properties of the Centyrin.
With a goal to achieve cell specific drug delivery, the team selects Centyrins tailored for cell surface receptors and format mono, bi or multi specifics in order to optimize binding and internalization of conjugated drug payloads such as oligonucleotides. Importantly the company owns the intellectual property rights to an extensive Centyrin IP portfolio.
Aro inked a deal last winter with Ionis Pharmaceuticals, Inc, which involved a potential payment of $1.4 billion for successful treatments by the gene therapy upstart. The biotech negotiated a license for a one of the programs that both Aro and Ionis are collaborating together on—it’s focused apparently on “a rare genetic muscle disease” apparently on its way to preclinical development this year.
About Aro Biotherapeutics
Founded in 2017, Aro Biotherapeutics has already closed $61 million, according to Crunchbase. Founder Susan Dillon, PhD, was the therapeutic area head of Immunology at Janssen before launching Aro. While the other founder Karyn O’Neil also has a long and impressive career in pharma including prominent positions in Johnson & Johnson.
Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of disease