Homology Medicines focuses on developing gene therapies to treat rare genetic diseases. Its pipeline is led by a gene therapy currently in Phase 2 development for adults with phenylketonuria (PKU). The company received a $60 million investment from Pfizer’s Breakthrough Growth Initiative, which the article says extended Homology’s cash runway to the third quarter of 2022. At the time of the report Homology had a market capitalization of around $500 million. The Pfizer investment ties Homology into a program that targets public and some private clinical-stage companies aligned with Pfizer’s therapeutic areas of interest. Future plans described in the article center on advancing its clinical-stage gene therapy program. Homology Medicines translates proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. Its AMEnDR™ (AAV-Mediated Editing by Direct Homologous Recombination) platform enables highly efficient homologous recombination-based in vivo genomic correction of disease mutations or precise insertion of therapeutic sequences. The platform does not use exogenous nucleases or promoters. The company plans to advance its lead development candidate, currently in preclinical IND-enabling studies for an inborn error of metabolism, toward clinical development. It also intends to progress AMEnDR™ into the clinic for rare genetic diseases. Financially, Homology closed an $83.5M Series B and has raised $127M since launching in 2016. The company is based in Bedford, Mass., and is led by CEO Arthur Tzianabos, Ph.D. Homology Medicines focuses on translating a proprietary, next-generation gene editing and gene therapy platform into novel treatments for patients with rare diseases. The platform harnesses the naturally occurring process of homologous recombination and is licensed exclusively worldwide from research by Saswati Chatterjee. The company was founded and incubated with a seed investment within the 4:59 Initiative, the creation engine of 5AM Ventures. Homology is led by CEO Arthur Tzianabos, COO Sam Rasty and CSO Albert Seymour and has assembled a board with investors’ representatives. Financially, the company completed a Series A financing to support its early development plans. The financing and seed backing position Homology to advance its platform toward therapeutic programs for rare diseases. Homology Medicines is a stealth startup launched with an initial $23 million investment disclosed in a regulatory filing. The company is being incubated by 5AM Ventures and is led by 5AM managing partner Kush Parmar. Listed on the filing is Dr. Richard Gregory of the Harvard Stem Cell Institute, whose research focuses on regulatory RNA and miRNAs implicated in disease. Article clues indicate the company may pursue RNAi/miRNA-focused research such as sequencing patient samples and characterizing miRNA expression profiles. The firm is Boston-based and currently operating in stealth; additional details about productization, clinical plans, or operating metrics were not disclosed. The report notes existing competitors in the RNAi/miRNA space, including Alnylam, Mirna Therapeutics, and Solstice Biologics.

Total raised

$210M

Funding rounds

4

Latest round

Equity

Latest activity

Jan 2021