ReCode Therapeutics Secures $260M Series B Financing to Advance Non-Liver-Targeted Gene Therapies Using Proprietary SORT-LNP Platform

Sep 21, 2023 07:20 CST Updated 07:20

ReCode Therapeutics

Gene Drug Developer

Solasta Ventures

Early-stage Venture Capital Firms

OrbiMed

Healthcare Investment Institutions

AyurMaya

Online Video Consultation Service Provider

Vida Ventures

A Life Sciences Investment Fund

MPM BioImpact

Healthcare Investment Institutions

Pfizer

Pharmaceutical R&D Developer

Sanofi

Pharmaceutical R&D Developer

Amgen Ventures

A venture capital firm based in California

September 21, 2023 / eMedClub News /--On September 19, 2023, ReCode Therapeutics announced the completion of a $50 million Series B extension financing.The cumulative amount of financing in Round B reached 2$600 million (approximately 1.9 billion RMB),This round of financing includes new investors BLV and Solasta Ventures.，Existing investors include OrbiMed Advisors, AyurMaya, Vida Ventures, MPM Capital, Pfizer, Sanofi, Amgen Venturesetc.。Proceeds from the financing will be used to advance ReCode Therapeutics' clinical development programs for primary ciliary dyskinesia and cystic fibrosis., and expand the company's proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) product line.

It is reported that ReCode Therapeutics is a comprehensive gene therapy company developing targeted, disease-modifying drugs for patients with life-limiting respiratory diseases. The company has built a broad and customizable non-viral LNP technology platform called Selective Organ Targeting LNP Delivery Platform (SORT-LNP). By introducing a fifth lipid component (SORT molecules) into LNPs, this platform creates its unique lipid formulation design, overcoming the limitations of traditional LNPs and enabling the targeted delivery of mRNA or gene-editing systems to specific tissues.

▲ Image Source: ReCode

Based on this technology platform, ReCode Therapeutics focuses on developing organ-specific delivery mRNA therapies, including lung-targeted, spleen-targeted, and liver-targeted therapies. The company has developed a rich pipeline, including treatments for cystic fibrosis and primary ciliary dyskinesia.The company's products utilize their proprietary LNP platform and nucleic acid technology to achieve mRNA-mediated replacement and gene editing/correction in target cells, thereby realizing the goal of disease treatment.

Revolutionary Therapies Enabled by Innovative Delivery Technologies

eMedClub

With its underlying innovative technology, the company was once selected for the Fierce15 list released by Fierce Biotech in 2022 and has gained favor from multiple multinational pharmaceutical companies and well-known investment institutions. In October 2021, ReCode Therapeutics completed an $80 million Series B financing round, led by Pfizer Ventures and EcoR1 Capital, with participation from Sanofi Ventures, the investment arm of Sanofi, among others. The funds raised will be used to advance the mRNA-based primary ciliary dyskinesia (PCD) program for cystic fibrosis. In June this year, the company announced the completion of a $120 million Series B+ financing round. Overall, the venture arms of major pharmaceutical companies such as Pfizer, Bayer, Amgen, and Sanofi participated in the company’s Series B financing round, showing optimism about the company’s prospects.

And canThe reason why it has gained favor from so many well-known enterprises and investment institutions may lie in its innovative delivery technology.PublicAs is known to all,DrugDelivery has always been referred to as the "last mile" of drug development, playing a pivotal role in the drug research and development process.EfficientThe drug delivery method can maximize the efficacy of the drug and improve treatment efficiency.The delivery vectors currently used in gene therapy or nucleic acid drugs mainly include lentivirus, adenovirus, adeno-associated virus (AAV), and LNP., but these delivery vehicles are not perfect; some have low delivery efficiency, some may integrate randomly into the genome, and some can trigger immune responses.AndCurrently, the tissue targeting of LNPs is mainly limited to the liver, and how to achieve targeting of non-liver tissues remains a challenge to be addressed.

The company's SORT-LNP platform holds promise to address this clinical pain point. This technology enables highly selective and organ-targeted delivery and can be used for drug delivery to extrahepatic tissues, a distinct advantage over general LNP technologies. Additionally, this delivery system can transport various types of genetic drugs, such as mRNA, siRNA, DNA, or hybrid drugs. Overall, this LNP technology further leverages the delivery advantages of non-viral vectors, potentially offering therapeutic solutions for a broader range of diseases. The SORT-LNP technology was published in Nature Nanotechnology in April 2020 and was also recognized by Nature as one of the "seven technologies to watch in 2022."

At present, the company's technology has made considerable progress. On March 15 this year,ReCode Therapeutics Announces First Healthy Volunteers Dosed in Phase 1 Clinical Trial of RCT1100RCT1100 is a "first-in-class" mRNA drug for the treatment of primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene. The company plans to submit an Investigational New Drug (IND) application for RCT1100 in PCD patients to the FDA in the second half of 2023 and to submit another IND for a CFTR mRNA therapy for the treatment of cystic fibrosis.

References:

1.https://www.vcaonline.com/news/2023091907/recode-therapeutics-announces-closing-of-extension-to-series-b-financing/

2.https://www.biospace.com/article/recode-scores-80-million-to-advance-mrna-based-cystic-fibrosis-pcd-programs-/?s=62

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