Laura Sanftner

Experienced nonclinical leader coordinating cross‑functional integration of nonclinical disciplines to deliver timely, fit‑for‑purpose safety assessments and clinical trial support. As the subject‑matter expert for siRNA nonclinical development, I lead Design Reviews and First‑Human‑Dose Safety Committee meetings to enable risk‑balanced decisions and ensure high‑quality regulatory dossiers (IB, IND/IMPD, DSUR/PSUR, RMP, NDA/MAA). Key responsibilities: -Lead nonclinical project teams and represent nonclincal on Global Project Teams in early phases -Plan and run nonclinical Design Reviews and initial Safety Committee meetings prior to FHD -Prepare and maintain project risk assessments and ensure follow‑up on activities, budgets and timelines -Coordinate complex cross‑functional and cross‑organizational tasks and documentation to support project progress -Conduct causality assessments and review safety literature for Safety Committee reporting -Advise on nonclinical strategy and contribute to major regulatory submissions and agency interactions -Present nonclincial topics to internal governance and externally at conferences -Mentor and supervise less experienced colleagues; drive knowledge sharing and best practices -Lead nonclinical due‑diligence teams and contribute to new project proposals and scientific publications

Develop and direct the toxicology strategy for development programs throughout the drug development lifecycle in conjunction with program teams Contribute to the preparation of nonclinical components of regulatory submissions (e.g., NDAs, INDs, IBs, and briefing packages) Represent Dicerna in Regulatory Agency interactions for toxicology-related issues Oversee identification, selection, and qualification of toxicology study vendors Review and provide feedback on study protocols and reports Collaborate cross functionally with Bioanalytical, Nonclinical ADME, Research, Regulatory, Clinical, and Manufacturing groups Communicate toxicology data to appropriate stakeholders including nonclinical functional areas, program teams, corporate partners, and executive leadership Oversee toxicology study monitoring team Manage budgets related to toxicology studies

Lead efforts to evaluate the pharmacology, metabolism, pharmacokinetics, and safety of novel small molecule therapeutics. Responsible for moving the Plexxikon’s late stage research molecules through IND-enabling safety activities and early clinical development by providing management of CRO partners for development activities.

Toxicologist and Neuropharmacologist • Nonclinical development of small molecules, plant-derived large molecules and gene therapeutics. • Managed outsourced and internal IND enabling studies: Tox, DMPK, and Safety Pharmacology. • Supervised a team of scientists and associates. • Successful IND and ex-US ethics committee submissions for Parkinson’s Disease, Pain, Addiction and Hemophilia indications.

• Collaborated with industry sponsor to evaluate therapeutic compounds effective in ameliorating vision impairment in in vivo models of Retinitis Pigmentosa. • Project leader to characterize of the molecular role neurotrophic factors play in the retinal disease. • Evaluated changes in expression of apoptotic genes in animal models of retinal degeneration over the course of the disease.

Dissertation titled “Development of AAV gene therapies for Retinal Degeneration" Received Graduate Student Teaching Award in 1998.