ReCode Raises $120 Million to Expand LNP Delivery Platform and Diversify Pipeline

Genetic medicines focused biotech ReCode Therapeutics raised $120 million in a new financing, bringing the total raised in its series B funding to $200 million, which it will use to expand its next-generation lipid nanoparticle drug delivery platform and pipeline of mRNA and gene correction medicines into the clinic.

Photo: Shehnaaz Suliman, CEO and board member, ReCode Therapeutics

New investors Leaps by Bayer and AyurMaya, an affiliate of Matrix Capital Management, co-led the new financing, with participation from Amgen Ventures. Pfizer Ventures and EcoR1 Capital co-led the initial series B financing, announced in October 2021, and included Sanofi Ventures, Orbimed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, Osage University Partners, Tekla Capital Management, Superstring Capital, and NS Investment.

Proceeds from the financing will be used to fund the diversification of ReCode’s pipeline into central nervous system (CNS), liver, and oncology indications, while continuing to advance the company’s lead mRNA programs for primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) into the clinic. The funds also will be used to advance development of ReCode’s platform to deliver a wider range of genetic medicine cargoes, including additional gene correction modalities and small interfering RNA (siRNA) therapies, to a wider range of target cell types in a predictable and programmable fashion.

ReCode’s selective organ targeting (SORT) lipid nanoparticle technology (LNP) platform is the foundation for its pipeline. ReCode said its SORT LNP platform is an innovation beyond the lipid delivery system used by the mRNA COVID vaccines and novel RNA and gene correction therapeutics.

LNPs are used to package and deliver genetic cargo such as mRNA. When delivered into the blood, first-generation LNPs primarily are taken up by the liver, which limits their utility for broad therapeutic applications. ReCode’s SORT LNPs are engineered with a biochemically distinct fifth lipid to help the body “sort” and direct the LNPs to other targeted organs such as the lung and spleen, with the ability to bypass the liver, if desired.

ReCode’s SORT LNP platform also has versability in both mode of administration (including IV, inhaled, subcutaneous, intramuscular, and intrathecal), and the diversity of genetic cargo that can be delivered (including mRNA, siRNA, DNA, gene correction components and mixed cargoes). Together, the company says these qualities offer vast opportunities to address a wide range of unmet medical needs through a precision medicine approach that delivers the right medicine to the right organs and cells using the optimal mode of administration.

“The successful administration of billions of doses of mRNA COVID-19 vaccines, and continued progress with novel RNA and gene correction therapeutics, have catapulted us into a new era of possibility for genetic medicines. We are harnessing that potential with our novel selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform, which is engineered with unique properties to enable delivery of genetic therapeutics directly to the organs and cells most impacted by disease, offering improved efficacy and potency. This potentially game-changing precision medicine technology creates vast possibilities for genetic medicines which have been limited by current technologies,” said Shehnaaz Suliman, CEO of ReCode Therapeutics.

The capability to precisely target specific organs and cell types is important for maximizing the efficacy of genetic medicines and limiting potential adverse effects. Additionally, as evidenced with the mRNA COVID vaccines and novel RNA and gene correction therapeutics, LNP packaging enables redosing. For people with genetic diseases such as CF that require potentially lifelong therapy, this means LNP delivered therapeutics can be administered repeatedly over time, potentially without significant immunogenicity.

In May 2022, ReCode presented preclinical data from its two lead programs in PCD and CF at the American Thoracic Society 2022 International Conference. These data demonstrated that selective organ targeting (SORT) LNP-formulated therapeutic candidates for PCD and CF can be precisely delivered directly to disease-relevant cells without significant exposure to other tissue, effectively releasing the encapsulated genetic cargo, and expressing the correct proteins at relevant levels. The company plans to advance to investigational new drug (IND) filings for PCD and CF in late 2022 and mid-2023, respectively.

In connection with the 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.

Author: Rare Daily Staff