The R&D of new drugs is inextricably linked to substantial financial backing. Within this framework, the role of venture capital firms is immeasurable. Today, we spotlight the renowned venture capital entity, ARCH.
ARCH Venture Partners, henceforth referred to as ARCH, was established in 1986. Over the years, it has positioned itself within the biotechnology sector by investing in several leading enterprises. These include the RNA therapeutics behemoth Alnylam Pharmaceuticals, gene therapy pioneer Bluebird Bio, CAR-T cell therapy innovator Juno Therapeutics, and base editing front-runner Beam, among others. ARCH stands out as one of the most prolific early-stage investors in the biotechnological domain. Beyond these well-known pharmaceutical companies, which burgeoning stars has ARCH supported?
Lyell Immunopharma: T-cell Therapy Development for Solid Tumors
Inaugurated in 2018 in San Francisco, California, Lyell Immunopharma is a preclinical stage biotechnological firm focused on the development of T-cell therapies for solid tumors. Distinguishing itself from conventional cell therapy enterprises, Lyell endeavors to fundamentally enhance the characteristics of T-cells essential for the treatment of solid tumor patients. This includes overcoming challenges such as T-cell exhaustion and loss of stemness, aiming to address barriers limiting cell persistence, reliability, and therapeutic efficacy.
The company surmounts these challenges through proprietary epigenetic and gene reprogramming technologies. Presently, Lyell has established two proprietary cell reprogramming technology platforms: Gen-R and Epi-R. Gen-R principally addresses T-cell exhaustion; Epi-R is utilized to generate T-cell populations characterized by enduring stemness.
Currently, Lyell is advancing new therapies based on these platforms, aiming to produce innovative, lasting, and potentially curative clinical approaches.
Arbor Biotechnologies: Gene Editing Company Co-founded by Zhang Feng
Established in 2016, Arbor Biotechnologies is a gene editing enzyme development company, co-founded by the esteemed scientist Zhang Feng. Arbor has developed seven gene editing enzymes capable of targeting over 90% of the genome and has identified more than 60 nuclease families and over 70 CRISPR transposases. The company's most advanced project is an in vivo gene editing therapy for Primary Hyperoxaluria (PH), ABO-101, currently at the IND enabling stage.
Verve Therapeutics: Pioneers in Base Editing Therapy
Conceived by the distinguished cardiologist Sekar Kathiresan, Verve Therapeutics is committed to developing preventive therapies for heart diseases through base editing technology. This approach envisages a one-time injection for the permanent prevention of heart diseases.
Verve's candidate, Verve-101, is the inaugural in vivo base editing therapy to enter clinical trials. It leverages lipid nanoparticles (LNP) to deliver the mRNA of an adenine base editor and gRNA targeting the PCSK9 gene. Upon injection, these nanoparticles are absorbed by liver cells, allowing the base editor to enter the nucleus and modify the specific sequence of the PCSK9 gene from A·T to G·C. This modification leads to the shutdown of gene expression, preventing the production of PCSK9 protein by liver cells, thus averting cardiovascular diseases.
In a phase 1b clinical trial, Verve-101 demonstrated a dose-dependent trend in reducing harmful low-density lipoprotein cholesterol (LDL-C) in patients with Familial Hypercholesterolemia (HeFH). A single administration of 0.6mg/kg of Verve-101 reduced LDL-C levels by 55%, with the effect persisting for over 180 days.
Verve plans to initiate a phase I clinical trial for another in vivo gene editing drug, Verve-101, in the first half of 2024, followed by a randomized, placebo-controlled phase II clinical trial for Verve-101 or Verve-102 in 2025.
hC Bio: Specializing in tRNA Drug R&D
hC Bioscience (hereinafter referred to as hC Bio) is a pharmaceutical R&D corporation concentrating on tRNA therapies aimed at addressing protein functionality disorders. tRNA (transfer RNA) constitutes an RNA molecule composed of 76-90 nucleotides, wherein its 3' end is capable of attaching a specific amino acid, a process catalyzed by aminoacyl-tRNA synthetase. Throughout the translation process, tRNA facilitates the transportation of the corresponding ...
(All Rights Reserved)