Primary Responsibilities
· Targets evaluation, target indications and competitive landscape assessment for RNA-based therapeutics, including siRNA, ASO and related disciplines
· Perform preclinical candidate screening and optimization via combined in vitro and in vivo assays, working withina cross-disciplinary group, including discovery chemistry, CMC and other relevant functions
· Determine mechanism of action of drug targets; design and execute proof-of-concept studies to validate targets together with internal and/or external partners
· Lead projects from inception to development candidate nomination
· Prepare key preclinical, clinical and regulatory documents, and patent application
· Effectively manage multiple programs and deliver quality result within sensitive timelines
· Comply with appropriate SOPs; if needed, train associates to meet requirements; present research findings to project teams, governance bodies, or external meetings
· Effectively communicate across all organizational levels
Qualifications
· PhD in biology, pharmacology, or related discipline with 4+ years of experience in pharmaceutical/biotech industry or academic settings. The level of position will be determined by previous experience and achievements.
· Experience in ASO/siRNA and/or gene/base editing is highly desired.
· Extensive experience conducting preclinical POC studies in the context of liver, extrahepatic tissues, such as nervous system (CNS or PNS), muscle
· Track record of independent research and creativity, as demonstrated by quality publications and/or patents
· Knowledge and ability to develop biochemical and molecular biology assays for RNA and protein quantification
· CRO and remote management of global resources, ranging from costing, execution, and interpretation of results in a timely manner
· Ability to work independently and collaboratively
· Outstanding organizational and time management skills and must be a self-starter with the ability to thrive in a multi-disciplinary, collaborative, fast-paced, and team-oriented environment.
· Excellent verbal and written communication skills
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