Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The company’s 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 Bio’s 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.
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Generation Bio is seeking an energetic and highly motivated Scientist/Senior Scientist to join the Cell Biology team within the Platform Research group. The position will play a key role in development and performance optimization of our non-viral gene therapy platform by developing and executing experiments aimed at deciphering the cell biological process involved in ctLNP uptake, endosomal escape and intra-cellular ceDNA transport. The ideal candidate will have a strong background in development and implementation of assays deciphering and manipulating the cellular immunological responses to ceDNA vector delivery, epigenetic modulation of ceDNA and assessment of its long-term expression in vitro and in vivo. The position will move seamlessly between leading lab-based work to design/implement assays in our state-of-the-art cell and molecular biology laboratories, providing scientific leadership roles in cross functional working groups and managing external studies with CROs and collaborators.
- Work in cross functional research teams to optimize ctLNP/ceDNA platform elements for desired long-term expression and immunologic profile of ceDNA
- Design and implement molecular biological tools like genome editing in vitro and in vivo
- Design, optimize and execute in vitro/in vivo assays to investigate potential immunogenic cell-autonomous and cell non-autonomous responses to cytoplasmic and nuclear ceDNA
- Develop and implement in vitro/in vivo assays to assess epigenetic modulations of ceDNA and their effects on long-term stability and expression
- Support development and evaluation of in vivo tools in appropriate animal disease models
- Summarize studies in written reports and oral presentations
- Interface externally with collaborators, and in management of research performed by CRO(s)
- Ph.D. in Virology, Molecular/Cell Biology, Immunology, Pharmacology or related field; minimum of 2 years of post-PhD experience preferred
- Experience in viral/non-viral gene therapy or a background in virology/ antiviral immunology highly desired
- Excellent lab technique and the ability to work independently to design experiments and analyze, interpret, and present data required
- Highly proficient in
- Cell culture (cell lines, primary cells) and cellular phenotypic and activity assays
- Genome editing assays
- Protein and DNA/RNA detection and quantification (RT-qPCR, (sc)RNAseq, ELISA, Western Blot, MSD, Octet)
- Epigenetic analysis assays (ChIP-seq, ATAC-Seq, Bisulfite sequencing)
- Cell-based high-content imaging (confocal imaging, IHC, flow cytometry, RNA/DNA (F)ISH)
- Demonstrated leadership capabilities in cross-functional integration and management
- Proactive thinking and ability to multi-task to support multiple projects across platform and disease biology pharmacology
- Ability to work in a highly collaborative, team-based environment a must
- Excellent written and verbal communication skills
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, religion, gender, national origin, 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.