Uniqure NV
Director, Regulatory Affairs, CMC
Uniqure NV, Lexington, Massachusetts, United States, 02173
uniQure is delivering on the promise of gene therapy -- single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in Huntington's disease and ALS, Fabry disease, temporal lobe epilepsy, and preclinical candidates.Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities.Position Summary:
uniQure is dedicated to bringing innovative therapies for unmet medical needs to patients. Each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility should be part of our day-to-day working life by (i) living up to high quality standards and (ii) recognizing the sense of urgency in areas of high medical need.Regulatory Affairs ensure our products meet the appropriate legislation to control their safety and efficacy from early development phases to the coordination of approval and registration. Regulatory Affairs are the crucial link between the company, the products, and the regulatory authorities, advising on and coordinating the development and approval of our products.The Associate Director RA-CMC is responsible for contributing to the design of a global regulatory CMC strategy for assigned products in line with the overall regulatory strategy and the direction of RA Leadership. The candidate is responsible for creating and implementing a global regulatory CMC plan and preparing content plans for regulatory CMC activities to ensure alignment with regulatory requirements and expectations, and that regulatory documents are prepared in high quality and submitted in a timely manner. The Associate Director closely collaborates with CMC, Manufacturing, Quality, and other RA functions to provide direction on the application and interpretation of global CMC regulations (primarily US and Europe) to support overall project goals. The Associate Director RA-CMC will also closely collaborate with the RA Product Lead to align with nonclinical and clinical development strategies.The position will also provide direction to internal customers, lead/participate in Agency engagement activities, and manage external CRO interactions.Responsibilities
Develop, communicate, and implement CMC regulatory strategy and ensure alignment with RA Leadership.Assure alignment with the overall global strategy through partnership and communication with other Regulatory Functions.Execute Regulatory CMC Strategy, activities, and deliverables for assigned products in alignment with the overall regulatory strategy and company goals.Plan for program deliverables in cross-functional meetings such as regulatory sub-teams, CMC Team meetings, and submission meetings.Participate in the Product team as the Regulatory CMC expert, working regularly with CMC, Manufacturing, and Quality functions to provide regulatory expertise for assigned products.Represent Regulatory Affairs in cross-functional teams and provide regulatory oversight and guidance to project teams on compliance matters independently.Lead and plan the content in the preparation of strategic regulatory documents throughout the product lifecycle.Lead and participate in regulatory CMC document planning, authoring, review, comments resolution, approval process, and delivery of submissions throughout the product’s lifecycle including briefing documents, IND/CMC, annual reports, and support the preparation of the team for negotiations with FDA, EMA, and other health agencies.Identify potential regulatory risks to the strategic/operational plans and propose options to mitigate risk.Advise CMC functions (for example, Process Development, Analytical Development, Manufacturing, and Quality Control) on the preparation and content of CTA/IMPDs, INDs dossiers to ensure timely delivery of final documents to be included in submissions.Identify potential regulatory risks to the CMC strategic/operational plans.Guide CMC stakeholders and project management on regulatory issues and impact assessment including CCRs and Deviations with the QA Team.Provide guidance to the CMC Team on process improvements for GMP processes in line with regulatory requirements.Conduct regulatory intelligence and follow evolving regulatory requirements, including ICH requirements, pharmacopoeias, guidelines, and regional requirements for assigned territories, and have an understanding of current global and regional trends in Regulatory Affairs and assess the impact on the CMC aspects of our development pipeline.Effectively communicate with internal teams and health authorities where applicable.Minimum Requirements
10+ years of Regulatory Affairs or related experience, Master or a higher scientific degree (PharmD, PhD).Experience with biologics/vaccines/advanced therapeutics is strongly preferred.Knowledge and practical understanding of ICH Guidelines regarding GMP/GCP, ICH Guidelines for CTD/eCTD, EMA and FDA Regulatory requirements, and the impact of these requirements on the business.Knowledge of Regulatory Strategies with a focus on the regulatory CMC Strategy.Strong technical writing ability and experience in writing submission documents and leading teams in the authoring process and preparation for Regulatory submissions (IND, CTA, BLA, MAA etc.) to the FDA and EU Regulatory Authorities.In addition to a RA CMC Background, understanding of pre-clinical/clinical regulatory requirements for drug development.Understanding the e-CTD specifications for electronic submissions.Ability to work in a matrix management and lead project teams through larger-scale projects and deliverables with efficiency.Respectful and collaborative team player both inside and outside the regulatory function.Ability to create, plan, and implement a submission process.Ability to rapidly identify and understand complex problems/situations; active participation in projects leading to solution development and implementation.Ability to implement corporate strategies globally, thinking creatively outside the box, while ensuring transparency and clear decision-making.Excellent oral and written communication skills which would be invaluable in communicating with global, cross-functional, cross-cultural, multi-disciplinary project teams including team members in country offices.Ability to successfully influence and negotiate issues within the organization and in a variety of settings.Works effectively across a broader and more senior set of stakeholders.Experienced in communicating vertically and horizontally within an organization and with necessary cultural awareness and sensitivity.
#J-18808-Ljbffr
uniQure is dedicated to bringing innovative therapies for unmet medical needs to patients. Each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility should be part of our day-to-day working life by (i) living up to high quality standards and (ii) recognizing the sense of urgency in areas of high medical need.Regulatory Affairs ensure our products meet the appropriate legislation to control their safety and efficacy from early development phases to the coordination of approval and registration. Regulatory Affairs are the crucial link between the company, the products, and the regulatory authorities, advising on and coordinating the development and approval of our products.The Associate Director RA-CMC is responsible for contributing to the design of a global regulatory CMC strategy for assigned products in line with the overall regulatory strategy and the direction of RA Leadership. The candidate is responsible for creating and implementing a global regulatory CMC plan and preparing content plans for regulatory CMC activities to ensure alignment with regulatory requirements and expectations, and that regulatory documents are prepared in high quality and submitted in a timely manner. The Associate Director closely collaborates with CMC, Manufacturing, Quality, and other RA functions to provide direction on the application and interpretation of global CMC regulations (primarily US and Europe) to support overall project goals. The Associate Director RA-CMC will also closely collaborate with the RA Product Lead to align with nonclinical and clinical development strategies.The position will also provide direction to internal customers, lead/participate in Agency engagement activities, and manage external CRO interactions.Responsibilities
Develop, communicate, and implement CMC regulatory strategy and ensure alignment with RA Leadership.Assure alignment with the overall global strategy through partnership and communication with other Regulatory Functions.Execute Regulatory CMC Strategy, activities, and deliverables for assigned products in alignment with the overall regulatory strategy and company goals.Plan for program deliverables in cross-functional meetings such as regulatory sub-teams, CMC Team meetings, and submission meetings.Participate in the Product team as the Regulatory CMC expert, working regularly with CMC, Manufacturing, and Quality functions to provide regulatory expertise for assigned products.Represent Regulatory Affairs in cross-functional teams and provide regulatory oversight and guidance to project teams on compliance matters independently.Lead and plan the content in the preparation of strategic regulatory documents throughout the product lifecycle.Lead and participate in regulatory CMC document planning, authoring, review, comments resolution, approval process, and delivery of submissions throughout the product’s lifecycle including briefing documents, IND/CMC, annual reports, and support the preparation of the team for negotiations with FDA, EMA, and other health agencies.Identify potential regulatory risks to the strategic/operational plans and propose options to mitigate risk.Advise CMC functions (for example, Process Development, Analytical Development, Manufacturing, and Quality Control) on the preparation and content of CTA/IMPDs, INDs dossiers to ensure timely delivery of final documents to be included in submissions.Identify potential regulatory risks to the CMC strategic/operational plans.Guide CMC stakeholders and project management on regulatory issues and impact assessment including CCRs and Deviations with the QA Team.Provide guidance to the CMC Team on process improvements for GMP processes in line with regulatory requirements.Conduct regulatory intelligence and follow evolving regulatory requirements, including ICH requirements, pharmacopoeias, guidelines, and regional requirements for assigned territories, and have an understanding of current global and regional trends in Regulatory Affairs and assess the impact on the CMC aspects of our development pipeline.Effectively communicate with internal teams and health authorities where applicable.Minimum Requirements
10+ years of Regulatory Affairs or related experience, Master or a higher scientific degree (PharmD, PhD).Experience with biologics/vaccines/advanced therapeutics is strongly preferred.Knowledge and practical understanding of ICH Guidelines regarding GMP/GCP, ICH Guidelines for CTD/eCTD, EMA and FDA Regulatory requirements, and the impact of these requirements on the business.Knowledge of Regulatory Strategies with a focus on the regulatory CMC Strategy.Strong technical writing ability and experience in writing submission documents and leading teams in the authoring process and preparation for Regulatory submissions (IND, CTA, BLA, MAA etc.) to the FDA and EU Regulatory Authorities.In addition to a RA CMC Background, understanding of pre-clinical/clinical regulatory requirements for drug development.Understanding the e-CTD specifications for electronic submissions.Ability to work in a matrix management and lead project teams through larger-scale projects and deliverables with efficiency.Respectful and collaborative team player both inside and outside the regulatory function.Ability to create, plan, and implement a submission process.Ability to rapidly identify and understand complex problems/situations; active participation in projects leading to solution development and implementation.Ability to implement corporate strategies globally, thinking creatively outside the box, while ensuring transparency and clear decision-making.Excellent oral and written communication skills which would be invaluable in communicating with global, cross-functional, cross-cultural, multi-disciplinary project teams including team members in country offices.Ability to successfully influence and negotiate issues within the organization and in a variety of settings.Works effectively across a broader and more senior set of stakeholders.Experienced in communicating vertically and horizontally within an organization and with necessary cultural awareness and sensitivity.
#J-18808-Ljbffr