Nonclinical Development & Translational Medicine Advisor

Nonclinical Development & Translational Medicine Advisor

Work Location: Primarily remote; on-site option available in Beijing

Job Overview

We are seeking an external, part-time Nonclinical/Toxicology & Translational Medicine Advisor to support our neurodevelopmental disorder programs based on AAV-delivered RNA-editing therapeutics, including novel AAV capsids. The Advisor will provide Sponsor-side guidance on key nonclinical development, toxicology, safety, and clinical-translation decisions.

The ideal candidate should have experience supporting first-in-class and novel-modality therapies with limited or no clinical precedent, together with hands-on experience in AAV programs, CNS translational medicine, CRO oversight, and U.S. and China regulatory interactions.

Beyond reviewing whether studies are appropriately designed and executed, the Advisor is expected to help the Company determine what studies are necessary, why they are needed, what level of evidence is sufficient, and how the resulting data can ultimately support FIH development and IND submissions—from an integrated Sponsor, regulatory, and clinical-translational perspective.

Job Responsibilities

• Provide Sponsor-side oversight of CRO-developed study protocols for CNS-administered AAV programs, including toxicology, biodistribution, and related nonclinical studies; participate in key CRO meetings and project milestones; identify potential scientific, safety, and regulatory risks and recommend appropriate mitigation strategies.

• Guide safety-risk assessment, biodistribution, and toxicology strategies for novel AAV serotypes/capsids; assess the scientific rationale and necessity of animal models and NHP study designs.

• Identify and address mechanism-specific risks associated with RNA editing, and provide guidance on appropriate nonclinical evaluation strategies.

• Provide translational medicine expertise on PK/PD, safety margins, FIH starting-dose selection, and dose-escalation strategies.

• Support regulatory interactions and submission strategy for FDA INTERACT, Pre-IND, and IND, as well as relevant regulatory engagements with NMPA/CDE in China.

Qualifications

• 10+ years of experience in nonclinical development, toxicology, or translational medicine for innovative therapeutics, with a strong track record in nonclinical development strategy, toxicology study design and review, and CRO oversight. Demonstrated ability to independently assess the scientific merit, necessity, and regulatory adequacy of nonclinical studies from a Sponsor perspective.

• Extensive hands-on experience in nonclinical development of AAV gene therapies. Experience with safety assessment of novel AAV capsids/serotypes is preferred.

• Strong understanding of the nonclinical development considerations for CNS-administered products. Experience with neurodevelopmental disorder programs is preferred.

• Experience supporting FDA IND, Pre-IND, and INTERACT submissions or regulatory interactions, paired with familiarity with relevant NMPA/CDE requirements in China.

• Demonstrated experience in translational medicine and FIH dose selection, including translating animal data into human clinical development strategies.

• Working knowledge of mechanism-specific toxicological risks associated with RNA editing, gene editing, or RNA therapeutics is preferred.

• Strong independent-consultant mindset, with the ability to align scientific and technical perspectives across internal teams and external partners, and efficiently deliver clear strategic recommendations and written assessments.