Slide 1: Innovation in gene synthesis & delivery
Innovation in gene synthesis & delivery July 2021
Slide 2: Presenters
Presenters Dr. Heikki Lanckriet, PhD. CEO & CSO David Roth CFO
Slide 3: Disclaimer
Disclaimer This document is intended for shareholders, prospective investors, partners and media audiences and no statements made here should be considered as claims for the success of our products, which are currently in the development phase. This document also may contain projections and/or estim
Slide 4: Overview
Overview ➢ Overview ➢ Insights ➢ Snapshot ➢ hpDNA™ ➢ Technologies ➢ DMD collaboration ➢ Product applications ➢ mRNA opportunity ➢ Timelines and objectives ➢ AAV opportunity ➢ Progress to date ➢ Q&A ➢ Near term objectives ➢ Timeline and vision ➢ Financial ➢ Financial Summary ➢ Funding Investor Presen
Slide 5: Overview
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Slide 6: Snapshot
Snapshot ➢ 4basebio is a specialist life sciences group focused on: ➢ supplying therapeutic synthetic DNA for gene therapies and gene-based vaccines ➢ providing solutions for effective and safe delivery of DNA/RNA based products to patients ➢ Serving a rapidly growing market, with the global cell an
Slide 7: Technologies
Technologies ➢ TruePrime™ 4basebio’s TruePrime™ DNA amplification technology is a primer free DNA amplification method using proprietary enzymes with high fidelity and processivity resulting in fast and accurate copying of DNA strands. This enables 4basebio to cost effectively produce large quantiti
Slide 8: osDNA
osDNATM ➢ What: ➢ Linear double stranded DNA with open ends ➢ Flexibility design on blunt or specific overhangs to suit needs ➢ Suitable for large and small constructs ➢ Suitable for incorporation of ITRs, poly A tails (validated to 120) ➢ Target applications: IVT mRNA, DNA vaccines and non-viral ve
Slide 9: Product applications - DNA
Product applications - DNA ➢ Uses of DNA ➢ Therapeutic agent in – non-viral gene therapies, DNA vaccines, gene editing ➢ Critical ingredient in production of mRNA vaccines, viral vectors, CAR-T ➢ Synthetic DNA ➢ Disruptive technology ➢ Protected by intellectual property ➢ USPs over plasmid DNA: ➢ Sa
Slide 10: Product applications - nanoparticles
Product applications - nanoparticles ➢ Use of viral and non-viral particles ➢ Packaging for delivery of gene therapies and vaccines ➢ Vector development ➢ Non-viral nanoparticles ➢ Developing non-viral delivery systems, generating new IP ➢ USPs over market-leading Adeno Associated Virus (AAV) delive
Slide 11: Timelines
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Slide 12: Progress to date
Progress to date ➢ Creating a business with valuable IP and essential development and manufacturing capabilities ➢ Established in house expertise to enable swift progress in 1. DNA process scaling 2. Nanoparticle development 3. mRNA in vitro transcription 4. AAV vector production ➢ Bring clean room
Slide 13: Near term objectives
Near term objectives ➢ Synthetic DNA ➢ Validate DNA across multiple applications including IVT mRNA, AAV, DNA vaccines, CAR-T, gene therapy and gene editing ➢ Scale and improve production processes for manufacture of DNA ➢ Adding IP (products and technologies) ➢ Non-viral nanoparticles ➢ Using techn
Slide 14: Timelines
Timelines ➢ Near term ➢ Longer term ➢ Make DNA and non-viral delivery systems available to prospective partners focused on ➢ Become a CDMO for the supply of synthetic mRNA vaccines, AAV vectors DNA across multiple applications ➢ Develop clean rooms and quality systems to be ➢ Progress multiple indic
Slide 15: Long term vision
Long term vision 4basebio UK Societas DNA and nanoparticle development 4basebio UK 4basebio Discovery DNA CDMO Non-viral delivery supplying DNA products and licensing technology, vectors in multiple markets developing therapies, service and applications work Investor Presentation
Slide 16: Financial
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Slide 17: Financial Summary
Financial Summary 2020 Results: 2020 2019 £’000 £’000 Revenue 462 202 Loss for the period (719) (423) Cash Balances 15,001 80 Net Assets 15,458 (1,890) ➢ Due to reverse acquisition accounting, the 2020 results reflect primarily 4basebio S.L.U. ➢ Results are not reflective of performance moving forwa
Slide 18: Group Funding
Group Funding ➢ No fund raising planned or foreseen ➢ Company is funded for foreseeable future through cash and debt facility ➢ Conceivable capital raise in due course to fund ongoing investment, e.g.: ➢ Large scale GMP facility project ➢ Progression of indications through IND into clinical trials ➢
Slide 19: Insights
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Slide 20: hpDNA
hpDNA™ ➢ Trueprime: High yielding, primer free DNA production, delivering DNA yields around 1g/l media ➢ Suitable for small and long constructs ➢ No sequence bias, able to amplify ITRs, tandem repeats, homo polymeric sequences etc ➢ High fidelity: NGS sequencing of 7.5B bases of Trueprime amplified
Slide 21: hpDNA
hpDNA™ ➢ Different constructs including constructs with ITRs and varying lengths of poly A tails have been produced using 4basebio Trueprime and hpDNATM technology ➢ The process yields a single target band on agarose gel ➢ No protein is detected on a Coomassie and silver stained SDS gel 1. 1,8 kb 2.
Slide 22: hpDNA
hpDNA™ ➢ Different DNA loads (2, 4 and 6 µg) and an ➢ No process enzymes visible in purified unpurified sample were run on and SDS page samples on silver stained SDS gel gel and stained with Coomassie. ➢ DNA in samples digested prior to gel loading ➢ No protein bands were detected at 6 µg DNA with D
Slide 23: DMD Collaboration
DMD Collaboration ➢ DMD is an X-linked inherited disease caused by mutations in the DMD gene, with an incidence rate of 1 in 3,500-6000 live male births. ➢ As a monogenic disease, DMD has long been regarded as amenable to a gene therapy strategy. ➢ Present AAV based DMD gene therapies focus on Exon
Slide 24: mRNA
mRNA ➢ mRNA is increasingly relevant for vaccines; e.g. Pfizer-BioNTech and Moderna COVID- 19 vaccines ➢ "Some vaccine makers will need hundreds of kilograms of DNA in the future. We estimate that the world's current supply of DNA is about 3 kilograms per year.“ (Jonny Ohlsen, Touchlight CEO, Forbes
Slide 25: AAV
AAV ➢ 2 gene therapies FDA approved ➢ More than ~150 therapies in clinical trials ➢ Sales value of DNA required for 1 patient dose can vary significantly; $75 - $10k 100% Similar levels of transfection obtained with hpDNA and GFP expression and MFI normalised to hpDNA 75% pDNA (blue bars) GFP expres
Slide 26: AAV
AAV 100% 100% hpDNA constructs show 95% 90% Normalised Full:Empty ratio consistently higher Full:Empty 90% 80% ratios than pDNA control Normalised TCID50 85% 70% (blue bars). 80% 60% 75% 50% 70% 40% TCID50 (Median Tissue Culture 65% 30% Infectious Dose) in HeLaRC32 60% 20% cells co-dosed with Ad5 sh
Slide 27: 2:4basebio
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Slide 28: Summary
Summary ➢ Well funded ➢ Strong scientific team ➢ Commercialising technology ➢ Valuation inflexion points over the coming 12 months ➢ Manufacturing capacity ➢ Commercial agreements ➢ Proof of concept on a multitude of applications and indications Thank you for listening Investor Presentation
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