Scientist/Senior Scientist, Preclinical Vector Development

Companie: Generation Bio
Tipul locului de muncă: Full-time

Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bios manufacturing scale to hundreds of millions of doses to support its mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.
We are a thriving, collaborative, creative community of 140 people with diverse talents, beliefs, and experiences headquartered in Cambridge, Massachusetts.
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SUMMARY:
Generation Bio is seeking an energetic and highly motivated molecular biologist to lead optimization of our ceDNA gene therapy in support of preclinical programs. In collaboration with Platform and Preclinical teams, the scientist will conceive and execute new construct designs to inform lead optimization across multiple disease indications. By leveraging knowledge of disease biology and fundamentals of mammalian gene expression, the scientist will customize ceDNA performance to meet individual program needs. This role is very cross functional with key interactions across diverse functions, including pharmacology, CMC, bioinformatics & molecular biology.
RESPONSIBILITIES:
* Work along-side pre-clinical teams to develop ceDNA for specific indications
* Design and generate constructs in support of pre-clinical development pipelines.
* Conceive and execute experiments to inform ceDNA optimization strategies
* Identify and incorporate platform learnings to meet disease specific therapeutic performance goals
* Oversee and manage construct databases and collections
* Work effectively within diverse cross functional teams to solve problems
* Manage CRO activities, including project design, timelines and quality of deliverables
QUALIFICATIONS:
* PhD in Molecular Biology, Virology, Mammalian Cell Biology or related discipline
* Minimum of 1 year of industry experience preferred
* Fluency with molecular biology techniques with a focus on application to mammalian systems
* In-depth knowledge of the molecular determinants of gene expression and its regulation, especially for mammalian systems
* Interest in disease biology and pre-clinical development
* Experience/ knowledge of AAV constructs, a plus
* Experience with bioinformatic analysis (e.g. Database curation/mining, Sequence analysis techniques), a plus
POSITION: Full-Time, Exempt
EEOC Statement: Generation Bio believes in a diverse environment and is committed to equal employment opportunity for all its employees and qualified applicants. We do not discriminate in recruitment, hiring, training, promotion or any other employment practices for reasons of race, color, gender, religion, national origin, gender identity, age, sexual orientation, marital or veteran status, disability, or any other legally protected status. Generation Bio will make reasonable accommodations for qualified individuals with known disabilities, in accordance with applicable law.

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