ReCode Therapeutics was formed around new science potentially enabling delivery of genetic medicines to various organs in the body, and the startup has encouraging data for its therapeutic candidates targeting the lungs. Now the biotech startup has $120 million to show that it can deliver on the technology’s promise in other organs and cell types in the body.
The financing, announced Wednesday, was co-led by Leaps by Bayer and AyurMaya, an affiliate of Matrix Capital Management.
Menlo Park, California-based ReCode uses lipid nanoparticles (LNPs) to deliver genetic medicines. As LNPs circulate in the blood, they preferentially seek out the liver. ReCode’s technology changes the way an LNP binds to proteins in the blood, removing the liver as a target. Scientists can then engineer the therapy to go to other organs in the body. This technology, initially developed at the University of Texas Southwestern, is called Selective Organ Targeting, or SORT.
ReCode’s two lead therapeutic candidates are in development for rare lung disorders. During the American Thoracic Society 2022 International Conference last month, the company presented encouraging preclinical data for both of them, one in development for cystic fibrosis (CF) and the other for primary ciliary dyskinesia (PCD). Results showed that the therapies were delivered to target tissues in the lungs without significant exposure to other tissues. Furthermore, these therapies were able to release their genetic cargoes and the therapies led to the expression of the correct proteins at what the company said were relevant levels.
LNP delivery brings some advantages compared with the engineered viruses used for some genetic medicines. These viruses introduce the risk of dangerous side effects. Also, the immune system sees these viruses as foreign and it produces antibodies against them. That means a patient can’t be given a second dose. David Lockhart, the former CEO and now chief scientific officer of ReCode, told MedCity News last fall that as a type of fat, lipids should not prompt the same immune response. That’s important for treating chronic diseases that require ongoing treatment.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Both the PCD and CF therapies are aerosolized formulations. Besides inhalation, ReCode says its technology can be used for genetic medicines given in ways that include intravenous infusion, subcutaneous or intramuscular injection, and spinal administration. Based on the encouraging preclinical results in the lung disorders, ReCode said it plans to file investigational new drug applications for the PCD drug in late 2022 and for the CF drug in mid-2023. The company also plans to apply the SORT technology to diseases in other tissue types. Specific diseases remain undisclosed but the company said it aims to address the central nervous system, the liver and cancer. In addition, ReCode said it will use its technology to deliver a wider range of genetic cargoes that could include gene correction medicines and small interfering RNA.
ReCode’s work has drawn interest from several large pharmaceutical companies. The company’s $80 million Series B round announced last October included cash from the venture arms of Pfizer and Sanofi. Amgen Ventures joined as a new investor in the financing announced Wednesday, which is an extension of the Series B round. With the latest financing, Alan Colowick, managing director of Matrix, and Rakhshita Dhar, senior director of venture investments health at Leaps by Bayer, will join ReCode’s board of directors.
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