Laura Jane Smith Email & Phone Number

Bedford, Massachusetts, United States

San Diego, CA, US

• Discovered and developed AAVHSC gene transfer and gene editing platform that led to initiation of Homology Medicines Inc.
• Key player in seed phase through facilitation of external technology verification and support of IP protection of novel AAVHSC platform.
• Instrumental in technology transfer of novel AAVHSC mediated gene transfer and homologous recombination editing platform from City of Hope to HMI.
• Hired during start-up phase to scientifically lead programs aimed to translate platform technology.
• Routinely presented key information on AAVHSCs internally cross-functionally as well as externally to potential and current scientific collaborators or partners.
• Played the role of “go to” expert for any questions or troubleshooting of platform technology or history throughout tenure at HMI.

• Strategized and executed on internal AAVHSC capsid development, IP generation and filing, and cross-functional capsid applications.
• Led new indication strategy for novel therapeutic area at HMI, selection process and exploratory indication POC using AAVHSC capsids.
• Supported business development through creation of decks highlighting key internal data and presented at technology deep dive meetings.
• Assessed future internal and external research AAV vector production strategy including costs, personnel, turnaround, production approach, quality and QC.
• Initiated cross-functional effort with Computational Biology team on continued development of AAVHSC capsid platform encompassing capsid and receptor/ligand/glycan modeling to support rational capsid engineering efforts.
• Point person for lab related issues which stemmed from HMI/OXBS transition.

• Spearheaded team on understanding of novel AAVHSC capsid tropism, which led to a first and corresponding author external publication addressing a key need in the translative AAV field (transaminitis).
• Investigated translational next steps of novel AAVHSC capsid and presented recommendations to upper management portfolio team.
• Designed and executed key in vivo studies to develop on AAVHSC platform using therapeutic payloads to address unknowns of technology.
• Initiated cross-functional effort for vector genome analysis of large NHP studies, data that allowed for next steps in therapeutic tissue targets of AAVHSC capsids.
• Managed research vector production relationship and goals with external OXBS team including timelines, queues, delivery and troubleshooting.

• Discovery Research
• Co-lead research on fast paced IND approved HMI-203 Hunter Syndrome gene therapy program starting from candidate nomination and including participation in cross-function development teams, IND study report generation.
• Led team and scientific research on therapeutic potential of AAVHSCs for ex vivo editing of HSCs and T-cells.
• Designed and led key objectives/experiments with several external partners in efforts to combine novel technologies with AAVHSC ex vivo editing potential.

• Scientific lead in key external partnership for AAVHSC mediated editing program for Sickle Cell disease encompassing presentations of key findings and decisions at JSCs and regular joint meetings, scheduling and.
• Collaborated cross-functionally on design and execution of in vivo and in vitro experiments to understand platform AAVHSC editing mechanism.
• Scientifically collaborated on multi-team efforts on internal IL2RG and F8 therapeutic editing programs.

• Program lead on internal HBB therapeutic AAVHSC mediated editing program, with delivery of primary cell in vitro and in vivo Sickle Cell model (CRO mediated) data and decision on program.
• Led research vector production team of 7 personnel for internal production, QC and delivery of novel AAVHSC gene transfer and editing vectors including prioritization of production queue with CSO.
• Collaborated cross-functionally on platform AAVHSC NHP biodistribution assessment, including performing tissue specific vector genome analysis.

Duarte, CA, US

• Investigated HSC and liver related therapeutic potential of novel AAVHSC mediated homologous recombination editing platform.
• Established novel AAVHSC mediated homologous recombination editing platform requires BRCA2 in vitro.
• Coordinated experiment and data analysis with third parties to validate technology as well as produced research grade vectors for collaborations.
• Drafted manuscript and figures for AAVHSC in vivo murine biodistribution.

Duarte, CA, US

• Investigated targeted integration of genes into hematopoietic stem cells using novel AAVHSC delivered genome editing tools in vitro and in vivo with external partner.
• Determined rAAVHSC hematopoietic stem cell transduction properties such as binding, nuclear entry and uncoating.
• Analyzed presence of human neutralizing antibodies to novel AAVHSCs in vivo and in vitro.
• Investigated ability of novel AAVHSCs to cross the BBB in murine model.
• Mentored summer students, graduate students and incoming research associates.
• Lead exploratory science classes for City of Hope-San Gabriel Valley Science Education Partnership Award Collaborative.

Duarte, CA, US

• Isolated novel AAV capsid sequences from human HSCs and created rAAVHSC vectors.
• Analyzed novel AAVHSC ability to transduce human HSC through in vitro HSC transductions and in vivo xenotransplants.
• Determined systemic distribution patterns of novel AAVHSCs through systemic and muscular injections.
• Related novel AAVHSC capsid changes with function through reciprocal mutagenesis and characterizing assays.
• Aided in characterizing hematopoietic stem cell transduction abilities of AAV tyrosine mutants.
• Mentored summer students, graduate students and incoming research associates.

Brentford, Middlesex, GB

• Trained to efficiency in GMP protocols for Media, Buffer, Equipment Prep and Purification.
• Wrote and coordinated uniformity in SOPs and Batch Records for Support Services and Purification.
• Led Buffer team through training, material procurement, and prepping and performing formulations, transfers and sanitations.
• Owned suite start-up projects for P&ID cartoons, Titrant Cans, HVAC protocol and IP21 tags.
• Researched, organized and cataloged metrology data for 150 pieces of equipment.
• Certified in Lean Six Sigma tools.

Doctor Of Philosophy (Phd), Biological And Biomedical Sciences

Certificate, Bioscience Management

B.S., Biology