Homology Medicines

Homology Medicines develops novel gene and gene editing therapies for the treatment of a range of genetic diseases starting off with monogenic diseases—diseases caused by defects in a single gene. The company  leverages its proprietary human hematopoietic stem cell-derived adeno-associated virus (AAVHSC) vector technology based on homologous recombination, a naturally occurring process to ensure DNA repair. This process deals with gene correction and does not require nucleases unlike common methods such as CRISPR/Cas9 that perform gene cutting. According to the company, the process enables precise on-target editing without unnecessary off-target DNA modifications. 

As of July 2022, Homology had seven programs in its pipeline, consisting of four gene therapies and three gene editing therapies. The company’s most advanced program is a gene therapy for adult Phenylketonuria (PKU)—a rare inherited disorder—in the Phase II clinical trials. As for its gene editing pipeline, as of 2022, one program was in the Phase I clinical trials for pediatric PKU and the remaining two were in the preclinical stages. 

Key customers and partnerships

The company has secured a number of big pharma partnerships that include Novartis in November 2017 to leverage Homology’s gene editing technology to develop therapies for select ophthalmic targets and a hemoglobinopathy disease and Pfizer in November 2020 with focus on the company’s PKU therapy, which also came with a USD 60 million equity investment. 

The company also launched a new AAV manufacturing and innovation business in January 2022 called ​​ Oxford Biomedica Solutions , in partnership with British company Oxford Biomedica. Homology owned 20% of the company’s stake, while Oxford Biomedica held the majority. 

Funding and financials

Homology Medicines, listed on the Nasdaq Global Select market since April 2018, raised gross proceeds of USD 50 million in its latest post-IPO public offering in April 2021. The proceeds were earmarked to advance its clinical development pipeline, expand its manufacturing capacity, and to fuel working capital expenses. For 2021, the company generated USD 34 million in collaboration revenue.