The Principal Scientist is responsible for leading Lacerta Therapeutics’ pipeline of programs related to Ataxia through preclinical and early clinical development stages. This individual will lead cross-functional teams dedicated to the translational development activities necessary to advance AAV gene therapy program(s) from research proof of concept to the end of Phase 1 clinical trials.
A successful candidate will be a hands-on, participative leader who will make significant contributions to therapeutic strategy and execution within a diverse R&D organization while exemplifying leadership and preclinical pipeline success.
Lead AAV gene therapy preclinical and early clinical development for Lacerta’s Ataxia-related programs, including identification of decision points and milestones and incorporating the required pre-clinical and validation studies, while managing risk to meet short and long-term objectives
Design, develop and execute IND enabling studies such as toxicology, pharmacokinetics and biodistribution studies for AAV constructs, independently and as part of a team
Assist in the support of regulatory requirements and the development of data packages for clinical trials
Collaborate with cross-functional teams to develop a working product profile, identify and evaluate appropriate animal models and novel targets for each program
Effectively coordinate and communicate program related activities to internal and external stakeholders
Work cross functionally with Lacerta’s QA/Regulatory functions, and the Finance Department to prepare & monitor budgets
Other role-related responsibilities as directed by the supervisor
PhD with post-training experience with 3+ years in repeat expansion diseases
3+ years of project team and/or leadership experience
Demonstrated track record in the AAV gene therapy preclinical development is preferred
PhD with post-training in ataxia-related repeat expansion diseases
experience in more than one functional area within the industry is a plus
Internal Number: 1
About Lacerta Therapeutics
Lacerta Therapeutics is a clinical-stage gene therapy company using a constellation of proprietary adeno- associated virus (AAV) vector technologies to develop treatments for central nervous system and lysosomal storage diseases. A resident company of UF Innovate | Sid Martin Biotech, the company is advancing clinical programs using proprietary capsid variants and a scalable vector manufacturing platform. Lacerta is currently focused on gene therapy solutions for Pompe Disease, Sanfilippo Syndrome Type B, Aromatic L-amino acid decarboxylase deficiency, Neurodegenerative Proteinopathies, Spinocerebellar Ataxias and Glioblastoma.