Valentina Galli

Leading Biology and Engineering Team of 6 Scientists/Research Associates, advanced and delivered key work packages focussed on developing groundbreaking viral and non-viral gene therapy systems (AAV and CRISPR/Cas9), bridging from in vitro to in vivo proof-of-concept, in collaboration with internal/external partners and CROs, managing budgets and reporting to stakeholders. • Spearheaded application of gene therapy products for heart diseases by leading collaboration with international academic partners to demonstrate successful biodistribution in large animals’ cardiac muscle, enabling Company’s outreach in new field. • Progressed product development project aimed at advancing neuron and immune cells specific delivery to inform target discovery efforts, successfully demonstrating improved biodistribution of CD4/CD8-targeted product upon systemic administration in vivo. • Delivered work packages essential to advance main platform and pipeline programmes, identifying gaps and promoting troubleshooting and problem-solving activities, enabling completion of production and analytical efforts under strict deadlines to contribute to major corporate goals.

Leading Biology and Engineering Team of 6 Scientists/Research Associates through major Company’s refocussing and restructuring, providing essential work packages aimed at viral and non-viral gene therapy systems (AAV and CRISPR/Cas9), translation from in vitro to in vivo proof-of-concept, coordinating and establishing collaborations with internal/external partners and CROs, managing budgets and reporting to stakeholders. • Identified need to assess in vivo delivery of targeted and non-targeted CRISPR/Cas9 genome editing product and demonstrated single-cell biodistribution in brain/CNS, obtaining essential results informing programme progression, tissue-specific targeting needs and choice of target indication. • Contributed to ideation, development and characterisation of immune-resistant AAV delivery system, currently being filed for IP (cited at co-contributor). • Led tech transfer of major assets post-IP acquisition to contribute to establishing AAV-mediated gene therapy programme.

Leading and supporting Cell Biology Team of 3 Scientists/Research Associates in delivering key projects to advance innovative product characterisation, analytics, improve PK and biodistribution, often in collaboration with other departments and external parties/CROs. • Successfully assessed feasibility of product nebulisation and led functional biodistribution study in lung to investigate potential alternative applications for current product (protein, oligonucleotide and AAV delivery system). • Identified method to increase biodistribution through engineering and modified product purification, collaborated with CMC department and external partner to demonstrate improved PK in vivo. • Increased product stability (50% improvement) by establishing and proposing alternative formulation. • Collaborated in project aimed at selecting lead candidate for functional loading of genome editing payloads into extracellular vesicles, successfully identifying key scaffolding proteins to maximise active molecule’s encapsulation and delivery in vivo. • Led collaboration with academic partner aimed at optimising vesicular loading of oligonucleotides, which resulted in publication in scientific journal. • Functioned as project manager for novel genome editing and research exploratory programmes, collaborating with Lead in establishing milestones, deliverables and managing budget, reporting directly to Corporate.

Established Cell Biology Team by identifying needs for developing product-specific methodologies and creating cross-departmental point of reference for product and process development, pitched the idea and gained buy-in from CEO, subsequently growing the group and recruiting scientific staff. • Developed method to characterise payload presence in vesicular drug delivery system (extracellular vesicles), directly influencing CMC department on manufacturing and process and analytical development. • Designed and developed a system to assay different cell sources for tissue-specific targeting and collaborated with various R&D and CMC groups to characterise and improve product and its purification. • Participated in projects aimed at engineering product to increase half-life in vivo and modify PK (publication in collaboration with academic partners). • Developed assays to demonstrate effective protein and siRNA cargo loading on extracellular vesicles, in the context of major collaboration with Pharma Company.

Contributed to Company’s pipeline projects, aimed at developing innovative delivery systems for ERT and oligonucleotides/small molecules therapeutics (through extracellular vesicles) for rare diseases. • Technical lead for in vitro research across the projects to establish efficacy and activity of ERT system in restoring lysosomal function in patient cells. • Designed and optimised biochemical and flow cytometry assays to prove product’s efficacy in vitro, in addition to establishing biochemical assays for proof of delivery in target organs in vivo. • Screened for lead single clone from a vesicle producer cell line, in order to establish a GMP bank of cells for manufacturing. • Collaborated in the design of animal studies to prove efficacy of engineered exosomes and their delivery to target organ, in addition to engaging with CMC to improve product purification and tech- transfer optimised assays for product analytics purposes. • As one of the first hired in the Company, contributed to establishing initial operations and participated in fundraising and partnering efforts by compiling reports, slide decks and SOPs for data room.

Successfully selected a small antibody (by phage display) specific for a conformational state of an endocytic protein, subsequently assessing the dynamics of its change of state in cells through total internal reflection microscopy. I established a novel technique to assess nanobody-antigen interaction of lipid-binding proteins. Identified protein that facilitates the uptake of artificial DNA-like gene-silencing probes (peptide nucleic acids), strongly contributing in the aim of characterizing their accessibility to cells, improving the possibility to find a controlled way for probe-delivery.

I was involved in a project on an intestinal glucose transporter, SGLT1, with the finality of finding a treatment for chemotheraphy-induced gastro-intestinal damage. I participated in demonstrating that glucose and analogues can protect the intestinal epithelium from chemotherapy-induced mucositis through the engaging of SGLT1.

I worked in human genetics with a focus on Amyotrophic Lateral Sclerosis, contributing in the research in finding genes involved in the familiar form of the disease.

I worked in a project researching post-transcriptional control of expression in bacteria.

Managed a furniture showroom, I was responsible for the product exposition and shop design, sales to the public and managed orders from the suppliers to the costumers.