Director/Senior/Executive Director, Gene Therapy Analytical & Formulation Development

At Locanabio, we are creating a new class of genetic medicines to treat a range of human genetic diseases with RNA targeted gene therapy. Our CORRECTx platform can precisely manipulate disease causing RNA by several mechanisms without DNA modification. We deliver our RNA manipulation  systems by gene therapy that potentially provides a durable effect with a 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. 

Position Summary:

This position will have leading responsibility for analytical and formulation development activities and staff within the larger CMC department to support Locanabio’s clinical pipeline of AAV-based genetic medicines products through accelerated rare disease drug development pathway. This important leadership role will be responsible for developing robust, compliant analytical control strategies, maintaining and continuously improving internal analytical development, formulation development and non-GMP testing capabilities, while also having responsibility for internal and external method development and qualification/validation of a wide-range of analytical methods at CDMOs in support of plasmid DNA release/stability testing, AAV drug substance and drug product extended characterization and cGMP release/stability testing. Other responsibilities include preparation/review of analytical CMC documentation, contribution to CMC regulatory strategy and authoring and review of analytical sections of regulatory submissions. You would have the opportunity to make significant contributions in our dynamic start-up environment where self-driven, enthusiastic employees thrive.

Responsibilities:

·       Using a combination of internal and external (CDMOs) resources, leverage strong technical expertise to drive the following activities for AAV drug substance and drug products throughout the development life cycle (FIH to commercial):

 - Design of control strategy and development/justification of material and drug substance and drug product specifications

 - Direct the method development, transfer, and phase-appropriate validation for release/stability methods, in-process methods, extended characterization methods, and starting materials and intermediate testing.

 - Compliant management of laboratory and OOS investigations, data trending and contribution to manufacturing investigations

 - Design and oversight of development studies for molecule/process/product characterization and comparability studies

 - Design and oversight of formulation development, robustness, clinical in-use compatibility and other related studies.

·       Develop compliant systems to manage stability programs for the entire life-cycle (concept to commercial) in compliance with ICH guidelines.  Activities include tracking stability test schedules at CMOs, summarizing and trending stability data, determining expiry/retest periods, and generating stability reports. 

·       Review and approve specifications, change controls and out of specifications reports.

·       Develop state-of-the-art AAV analytical and formulation platforms that can be leveraged for developmental efficiency across the pipeline programs.

·       Analytical development budgeting and resource management, including CDMO proposals/invoicing, as well as management of employees, contractors and consultants.

·       Provide testing support for internal process development and toxicology vector production activities from our internal pilot production lab.

·       Participate in and lead 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.

·       Stay abreast of evolving Gene Therapy Regulations and Guidance, and contribute to development of CMC Regulatory strategies, including strategies for and participation in Health Authority submissions, consultations/meetings and responses.

·       Author and/or review appropriate development reports and related sections of Regulatory filings, such as INDs, IMPDS, CTAs, BLAs, etc.  Responsible for the quality of development studies, reports, and regulatory documents.

·       Contribute to vendor selection including drug substance/product and analytical CDMOs.

·       Contribute to development and continuous improvement of Locanabio quality systems, including authorship/revision of SOPs and the design and implementation of lean, phase-appropriate systems to support fast-moving IND drug development programs.

·       Support Quality Assurance on quality system and compliance activities, including audit functions, as needed.

·       Adhere to internal and external regulatory (ICH, FDA, EMA, etc.) and GMP requirements.

·       Extensive knowledge of cGMP, ICH and FDA guidelines, and familiarity with ex-US Regulatory guidelines.

·       Proficient in standard analytical techniques used in the biopharmaceutical industry

·       Competency in Microsoft Word, Excel, and PowerPoint; familiarity with Microsoft Project.

·       Travel: Up to 15% – occasional international travel to partner and/or contractor facilities.

Personal Competencies:

·       Ability to effectively manage multiple responsibilities, tasks, and projects simultaneously in a dynamic, fast-paced environment.

·       Proven leadership experience with recruiting, managing and developing teams.

·       Excellent oral and written communication skills.  Ability to communicate clearly, and to present experimental results and analysis, and persuasively present recommendations.

·       Demonstrated ability to independently develop solutions to complex problems.

·       Effective interpersonal skills.  Ability to work in a team-oriented, cross-functional environment, and effectively manage diverse personalities and styles.

·       Self-motivated and results-oriented with initiative to complete tasks independently.

·       Capacity to organize assignments, work within deadlines.

·       Team player intensely committed to getting the job done well in a dynamic 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.

Requirements:

·       PhD in bioanalytical chemistry or relevant field with a minimum of 12 years of relevant industry experience in analytical development for gene therapy, or MS with a minimum of 15 years of relevant industry experience.  Strong preference for at least 5 years of experience managing outsourced analytical activities at contract organizations.

·       Demonstrated technical proficiency in a wide variety of analytical techniques involved in the analysis of viral vectors, including but not limited to HPLC/UPLC, AUC, SEC, AF4, HIAC/MFI, LC-MS, NGS, DLS CE-SDS, PCR, ELISAs, cell-based potency/infectivity assays, micro/sterility methods and all compendial methods. Experience with qualifying/validating these methodologies in a QC setting. 

·       Extensive experience with assay development, qualification/validation and method transfer at external CROs/CMOs, with solid knowledge of cGMP practices for analytical laboratories.  Prior experience in Quality Control, Quality Assurance and/or Regulatory Assurance roles is a plus.

·       Broad, and in-depth expertise in characterization methods used in the development of biologics, including but not limited to, Mass Spec, Peptide Mapping, PTM, primary/secondary/tertiary structural analysis and structure-function relationship.

·       Expertise in setting phase-appropriate process and product specifications.

·       Demonstrated expertise with comparability strategies for biologics due to manufacturing changes; strong preference for specific comparability experience with viral vectors.

·       Significant understanding and experience in strategic and tactical elements of transitioning from early-stage to late-stage development of biologics.

·       Direct experience preparing and reviewing CMC documentation for regulatory filings and required.  Experience meeting and interacting with FDA and other ROW Regulatory authorities is a strong plus.

·       Experience with sterile, parenteral /injectable products.

·       Experience in project management and team facilitation 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 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.

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Salary : $200,000 - $300,000