DYNE-251
Dystrophin-targeted conjugate. Completed the nonclinical package substantiating safety on the program's timeline.
IND cleared
The safety case a regulator cannot send back.
Marc Roy, PhD — Nonclinical Safety & Preclinical Development
Fifteen years moving genetic medicines from laboratory concept to cleared regulatory submission — across siRNA, exon-skipping oligonucleotides, antibody-oligonucleotide conjugates, AAV, and LNP-delivered mRNA. Multiple INDs filed and cleared without preclinical inquiries.
15+Years in nonclinical safety and preclinical development
$25MSafety portfolio held at Sarepta Therapeutics
7Toxicologists and immunologists led
4Lab-developed-test reports accepted by the FDA
The work
Will this molecule hurt someone, and can we prove what we believe about it to a regulator?
That is the question a nonclinical safety function exists to answer. Answering it well means designing the right studies, holding contract laboratories to submission standard, reading data rather than receiving it, and writing a submission that anticipates the reviewer's questions before they are asked.
Marc has done this across large-pharma drug safety research and venture-backed genetic medicine companies — most recently at Sarepta, Strand, Design, and Dyne Therapeutics, and earlier across eleven years at Pfizer in molecular pathology and investigative toxicology.
Program ledger
Named compounds carried through nonclinical safety
DT-168
Novel trinucleotide-repeat-binding construct with no established regulatory precedent to draw on.
IND cleared — no preclinical inquiries
DT-216
Held safety and pharmacology accountability across the preclinical portfolio.
Safety & pharmacology owned
SRP-1001
Study strategy and endpoint selection through to data interpretation and internal risk position.
Nonclinical safety owned
SRP-1003
Run in parallel with AAV work, requiring separate toxicology judgement per modality.
Nonclinical safety owned
SRP-9010
Authored the preclinical safety content underpinning the program's regulatory progression.
Regulatory advancement supported
STX-001
One of four candidates on a lipid-nanoparticle mRNA platform.
IND documentation authored
STX-003
Design choices matched to both the scientific question and regulatory expectation.
GLP & non-GLP program delivered
STX-004
Platform-level safety agenda rather than candidate-by-candidate response.
GLP & non-GLP program delivered
STX-005
Submissions kept to schedule across the candidate set.
GLP & non-GLP program delivered
Drag, or use the arrows — 10 programs
Modality breadth
Seven modalities, each with its own failure mode
siRNA
Nonclinical safety ownership across siRNA candidates, including study strategy, endpoint selection, and the internal risk positions that follow from the data.
Exon-skipping oligonucleotides
Safety strategy across a $25M exon-skipping and AAV portfolio, setting what was investigated, what was monitored, and what warranted escalation.
Antibody-oligonucleotide conjugates
Conjugate toxicology where antibody and oligonucleotide liabilities interact rather than sit independently — the basis of the DYNE-251 clearance.
Antibody-drug conjugates
Molecular pathology lead to Pfizer's global investigative ADC team, including patient-selection assay development and validation.
AAV gene therapy
Program-level safety across AAV delivery at Sarepta and gene therapy for neurological disease at Novartis.
LNP-delivered mRNA
Platform safety agenda for four lipid-nanoparticle mRNA candidates, from study design through IND documentation.
Small molecules
Mechanistic investigative toxicology, including kinase-inhibitor-induced hyperglycemia across a six-month rat study.
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The toxicology of these modalities does not transfer cleanly between them. Conjugates, vectors, and delivery systems each fail in their own way, and recognising which failure mode is in front of you is not something acquired quickly.
In practice
Where the work happens
Cross-functional review — translating safety findings into decisions
Data interpretation — reading a dataset rather than receiving it
Mechanistic investigation underpinning safety position
GLP-standard facilities and outsourced study oversight
CRO deliverables held to submission quality
Study execution across internal and external laboratories
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Currently
Open to Head of Nonclinical Safety, VP, and preclinical development leadership roles.
Unconstrained in location for the right organization and the right science.