At Locanabio, we are creating a new class of genetic medicines to treat a range of human genetic diseases by delivering RNA binding proteins and effector domains that can precisely manipulate disease causing RNA by several mechanisms without DNA modification. We deliver our RNA manipulation systems by gene therapy which provides a durable effect with one-time treatment. The broad range of RNA manipulations that are possible with our approach provides a unique platform to address many diseases beyond which is possible with current therapies. The wide range of potential indications is supported by the modularity of our RNA-targeting gene therapies which provide exquisite control of specificity and activity.

 

Overview:

 

This scientist will design, execute, and manage activities related to biophysical assay development, validation, and implementation for lot release testing and extended characterization of Locanabio’s innovative AAV gene therapy products. The scientist must be well versed in all aspects (technical, compliance, regulatory) of release and characterization testing for viral vector biophysical 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 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. 

Responsibilities:

• Design, execute and manage assay development, transfer, and validation for AAV release/stability, in-process, extended characterization, and starting materials and intermediate testing. 
• Strong technical expertise and experience in methods for assessing viral capsid protein content, composition, quality 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.

Requirements:

• PhD in Biological Sciences or relevant field plus minimum of 7 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 ELISA, HPLC, CE, AUC, DLS, LC-MS, FFF-MALS are required.
• 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.

Personal Competencies:

• 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.