Lead Research Organisation: University of Oxford


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Technical Summary

While accelerated development of a vaccine(s) for Covid19 is essential, in parallel rapidly
developing a diverse armoury of effective anti-viral therapies is of crucial importance. Here, we
seek seed funds to initiate PHASE ONE of the development of the FIRST ANTISENSE
OLIGONUCLEOTIDE (ASO) THERAPY for Covid19, rapidly undertaking ASO screens on prioritised
SARS-CoV-2 RNA targets, in vitro and in vivo efficacy assays and preliminary safety studies to
identify lead ASOs and to generate a preliminary data package to support a larger funding
application to MRC/UKRI. The initial team comprises Wood (University of Oxford and Nucleic Acid
Therapy Accelerator, Roux (Nucleic Acid Therapy Accelerator) and Bauer (Francis Crick Institute). In
PHASE TWO, we will iterate lead ASOs with emerging SARS-CoV-2 genomic and RNA structural
biology data (Bauer) to complete final selection of an optimised ASO and then generate a
complete preclinical dataset (comprising PK, PD, dose ranging, biodistribution, safety) to inform
clinical trial design and the final clinical development plan. We will then work with our industrial
partner Wave Life Sciences (Boston, USA) to rapidly deliver the scaled up clinical development of
the lead ASO candidate, comprising appropriate regulatory guidance, non-human primate safety
study package and GMP manufacturing. Thus, building on well-established ASO platforms, SARSCoV-
2 genomic expertise, and industrial drug development capabilities, our overall goal is to
accelerate development of THE FIRST ASO THERAPY to a first-in-man Phase I clinical trial in
Covid19 patients in less than 12 months.


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