London-based AviadoBio has been granted FDA’s and European Commission’s (EC) orphan designation for its frontotemporal dementia (FTD) gene therapy (AVB-101). AVB-101 is a one-dose adeno-associated virus (AAV) gene therapy for FTD patients with mutations in the progranulin gene (GRN), which accounts for up to 40% of the FTD cases. It aims to slow down the disease progression by delivering a healthy copy of GRN through the central nervous system. Clinical trials in UK will start late 2022. AviadoBio raised $80 million investment in December 2021 and the round was led by New Enterprise Associates. FDA grants ‘Orphan designation’ to drugs aiming to treat conditions affecting fewer than 200,000 people in the US, whereas EC grants it for those intending to treat diseases affecting less than five in 10,000 people in EU. FTD is the second most common form of dementia in adults under 65.
Quell Therapeutics and Ncardia’s Cellistic have signed an agreement to develop an iPSC-derived allogenic T-regulatory (Treg) cell therapy platform. The collaboration aims to exploit Cellistic’s development and manufacturing capabilities to help advancing Quell’s pioneering autologous CAR-Treg cell therapies. Quell Therapeutics is one of the top 10 university spin-outs by investment volume (£117m), and raised $156 million series B in November 2021. Their lead candidate (QEL-001, LIBERATE study) is targeting liver transplant rejection, a process driven by immune dysregulation.
Charles River Laboratories (CRL) and Valo Health have launched Logica, an AI-powered drug discovery platform to deliver optimised preclinical assets. The partnership aims to leverage Opal, Valo’s computational platform, and CRL preclinical expertise. Logica’s Advanceable Lead (Logica-AL) and Logica’s CAndidate (Logica-C) utilise Valo’s proprietary predictive modelling, synthesis and chemical design and high-throughput screening, and will be exclusively available from CRL. The aim is to deliver drug candidates that are ready for the IND-enabling studies and have undergone all required safety and efficacy checks.
Spanish-owned biotech Quibim has selected Cambridge for its first expansion to the UK. The decision was taken together with the Growth Works Business Service and its investment arm Locate Cambridge. Quibim, a whole-body medical imaging startup, had to pitch to a panel formed by Start Codon, Illumina, Eastern Academic Health Science Network(AHSN), and a member of the Department of International Trade’s Global Entrepreneurship Programme. Quibim has developed the Quibim Precision (QP) suite and their most advanced system, QP-Prostate, was found to fit into the NHS’ strategic plan.
Nottingham-based Sygnature Discovery has acquired Peak Proteins, a company set up n 2014 by a group of AstraZeneca’s protein scientists and structural biologists. It represents the 4th acquisition in the last four years and aims to enhance Sygnature’s biophysics and high-throughput capabilities.
CN Bio has launched PhysioMimix, an in-a-box/in vitro model for non-alcoholic steatohepatitis (NASH). The company aims to accelerate the incorporation of single and multi-organ microphysiological systems (MPS) into the drug discovery workflow. This would provide researchers with in-house capabilities to analyse disease-driving mechanisms, safety and efficacy profiles, etc. PhysioMimix allows for formation of 3D tissue structures with primary human hepatocytes, stellate and Kupffer cells.