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Ionis Pharmaceuticals

2855 Gazelle Court, Carlsbad, CA, 92010, United States

Overview

Ionis Pharmaceuticals is a pharmaceutical company. It specializes in RNA-targeted drug discovery and development. Ionis has developed antisense technology, an efficient, broadly applicable drug discovery platform that can treat diseases where no other therapeutic approaches have proven effective.

Total investments
2
Lead investments
1
Investments · 12mo
0
Active investors
8

Sector focus

  • Biotechnology
  • Health Care
  • Medical
  • Pharmaceutical
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Investment portfolio

  • Aro Biotherapeutics

    Participated · Series A · Jan 2021

    Aro Biotherapeutics is a Philadelphia-based clinical-stage biotechnology company pioneering tissue-targeted genetic medicines with a proprietary Centyrin protein platform. The company develops Centyrin-siRNA conjugates to enable efficient, tissue-specific delivery of siRNA and is building a wholly owned pipeline of therapeutic candidates. Its lead program, ABX1100, targets Pompe disease via a CD71-binding Centyrin conjugated to an siRNA against Gys1 and demonstrated durable reductions in Gys1 mRNA in muscle tissues with a favorable GLP toxicology safety profile. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the FDA, and Aro has initiated a first-in-human study with initial data expected in 2024. The Series B financing will be used to advance ABX1100 and to support discovery of additional Centyrin-siRNA conjugate programs focused on autoimmune disorders. The new funding of $41.5M positions the company to continue clinical development and pipeline expansion. Aro Biotherapeutics is developing Centyrin-conjugated RNA therapies, a proprietary platform that uses engineered Centyrin proteins to target RNA medicines to specific tissues and receptors. Centyrins are described as small, exceptionally stable, engineered human proteins designed to target receptors on specific cells and deliver complex drug payloads to disease sites. The company is advancing a wholly-owned pipeline and is working with industry partners to leverage Centyrins for tissue-specific targeting across a diverse set of diseases. Aro plans to use the Series A proceeds to advance its lead therapeutic candidates into clinical development, with an initial focus on rare genetic and immune disorders. Leadership highlights the potential for improved efficacy and safety by enabling more efficient and precise delivery of RNA drugs to extra-hepatic tissues. Aro was founded in 2018 by Susan Dillon and Karyn O'Neil and is based in Philadelphia. Aro Biotherapeutics is developing Centyrins, small, ultra‑stable, highly soluble engineered proteins designed to simplify antibodies and enable new mechanisms of action for oncology and immunology. The company holds an exclusive worldwide license to Centyrin therapeutics discovered by co‑founder Karyn O’Neil at Janssen and is building a wholly owned pipeline. Aro’s lead program is a bi‑specific Centyrin in late‑stage lead optimization for advanced non‑small cell lung cancer; a second program aims to create a Centyrin‑siRNA conjugate to deliver nucleic acid drugs to tumors and immune cells. Centyrins have been optimized for potent anti‑tumor activity and efficient manufacture in E. coli, and Aro intends to leverage them to address targets considered ‘undruggable’ while improving efficacy and safety profiles. The company has recruited an experienced scientific and executive leadership team co‑founded by Sue Dillon and Karyn O’Neil and headquartered in the Pennovation Center in Philadelphia. Aro established operations with a $13 million start‑up investment from Johnson & Johnson Innovation (JJDC) and BioMotiv to fund development and commercialization of its Centyrin programs.

  • Empirico

    Led · Series A · Jan 2020

    Empirico’s core product is its Precision Insights Platform, a human genetics‑focused discovery platform that combines a large curated biological dataset with statistical and machine‑learning algorithms to generate and prioritize therapeutic hypotheses. The platform pairs in silico target discovery with experimental validation to elucidate mechanisms by which genetic variation impacts disease and to identify appropriate therapeutic modalities. Empirico has active preclinical programs across immune, dermatological, cardiometabolic, and ophthalmic indications based on targets from the platform. The company announced a strategic three‑year collaboration with Ionis to identify targets amenable to antisense oligonucleotide therapeutics and to incorporate human genetics evidence into Ionis’s ongoing programs for target validation, indication and biomarker selection, and patient stratification. Financially, Ionis made a $10 million equity investment in Empirico and the company closed a $17 million Series A‑2 financing; Empirico also raised a $12.5 million Series A in November 2018. Under the collaboration Empirico is eligible to receive over $620 million in clinical, regulatory and commercial milestones plus royalties, and may receive additional near‑term commitments from Ionis of up to $30 million tied to operational and preclinical milestones.

Team

  • Eugene Schneider

    EVP and Chief Clinical Development Officer

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  • Eric Bastings

    Vice President, Development Strategy

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  • Meghdad Rahdar

    Assistant Director

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  • Brent Golec

    Executive Director, Pharmaceutical Development

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