Senior/Principle Scientist, Gene Therapy Bioassay Development
At Locanabio, we are creating a new class of precision medicines to treat a range of human genetic diseases. Locanabio's RNA-targeting gene therapy platform allows us to precisely address these mutations on the level of RNA. By targeting RNA, our approach avoids the risk of off-target effects in DNA and is suited to address many diseases linked to dysfunctional processing of mRNA. The wide range of potential indications is supported by the modularity of our RNA-targeting gene therapies which are composed of interchangeable components that provide exquisite control of specificity and activity.
We are seeking an outstanding candidate to join our development team. You would have the opportunity to make important contributions in our dynamic start-up environment where self-driven, enthusiastic employees thrive.
Senior/Principle Scientist, Gene Therapy Bioassay Development Position Summary: This scientist will design, execute, and manage activities related to biological assay development, validation, and implementation for lot release testing and characterization of Locanabio’s innovative AAV gene therapy products. The scientist must be well versed in all aspects (technical, compliance, regulatory) of cell-based functional potency assays. The candidate must be able to conduct these activities in-house, and/or at Contract Development and Manufacturing Organizations (CDMOs) and Contract Testing Organizations (CTOs). This individual will also support other analytical development activities outside of bioassay development to help facilitate the progression of Locanabio’s clinical pipeline programs. Additional responsibilities will include preparation and review of analytical CMC documentation, contribution to authoring and review of analytical sections of regulatory submissions.
- Design, execute and manage biological assay development, transfer, and validation for release/stability, in-process, extended characterization, and starting materials and intermediate testing. Strong technical expertise and experience in cell-based potency assays is required; experience in a Gene Therapy and/or Gene Editing context is a strong plus.
- Execute and support development studies for molecule/process/product characterization and comparability studies.
- Draft, review and approve SOPs, records/forms, protocols, specifications and reports for transfer to internal and/or external QC lab(s).
- Lead technical diligence efforts in support of the selection of CDMOs/CTOs for biological potency assay development/testing or other analytical testing as needed
- Participate in cross-functional teams, including partner and CDMO interactions. Facilitate coordination of analytical activities with research, process development, manufacturing, quality, regulatory, and clinical supply functions as necessary.
- Support Quality Assurance on quality system and compliance activities, including audit functions, as needed.
- Assist in the preparation of CMC regulatory submissions, including providing data summaries and trend analysis.
- Adhere to internal and external regulatory (ICH, FDA, EMA, etc.) and GMP requirements to develop and establish analytical policy and process to facilitate department operations.
- Travel: Up to 25% – occasional international travel to partner or contractor facilities.
- Ability to effectively manage multiple responsibilities, tasks, and projects simultaneously in a dynamic, fast-paced environment.
- Excellent oral and written communication skills. Ability to communicate clearly, and to present experimental results and analysis, and persuasively present recommendations.
- Effective interpersonal skills. Ability to work in a team-oriented, cross-functional environment, and effectively manage diverse personalities and styles.
- Self-motivated, independent, and results-oriented with initiative to complete tasks independently.
- Intensely committed to success and getting the job done well in a team environment.
- Strong analytical and organizational skills; highly attentive to details, but also the ability to think in terms of big picture strategy.
- Play a key role in meeting cross-functional goals through commitment, quality standards.
- Contribute to a work environment that fosters professionalism, mutual respect, teamwork, and collaboration.
- PhD in Biological Sciences or relevant field plus minimum of 10 years of experience in the biotechnology or pharma industry, with a primary focus on analytical development for biologic products (MS/BS with more extensive relevant experience also considered)
- In-depth knowledge and experience in the biotechnology or pharma industry, with a primary focus on the method development, validation, and application of analytical methods for product development, characterization, and QC testing.
- Extensive experience and expertise in analytical techniques routinely used to test vector products, including cell-based assays, enzymatic assays, flow cytometry, ELISA, and DNA sequencing/PCR. Expertise in HPLC/UPLC, and LC/MS are a plus.
- Demonstrated effective organizational, project management and problem-solving skills. Must be an effective leader to plan and to direct scientists to achieve deliverables and meet timelines.
- Solid understanding and familiarity with AAV vector process development, characterization, validation, and production is a strong plus.
- Experience with managing CDMO/CTL relationships and projects
- Sound understanding and demonstrated application of statistical methods/tools (e.g. SAS, JMP)
- Knowledge of US and European Pharmacopeia methods and requirements
- Knowledge of FDA, EMA, GMP, WHO and ICH regulatory requirements
- Excellent oral and written communication skills
Locanabio is dedicated to fostering a workplace that keeps our team inspired and engaged. We offer an inclusive company culture where employees feel respected and valued.
Locanabio offers a competitive benefits package including medical, dental and life insurance benefits for employees and their families. We also offer a 401(k) plan with a matching contribution and stock options. Locanabio is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation, veteran status, or any other protected categories.
Send your cover letter and resume for immediate consideration.