uniQure
Director, Regulatory Affairs, CMC
uniQure, Lexington, Massachusetts, United States, 02173
Director, Regulatory Affairs, CMC
Location:Lexington, MAEducation level:Master or PhD
Job category:Regulatory AffairsTarget start date:12/1/2024
Work Location:HybridShift:1st
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 bring innovative therapies or unmet medical needs to patients. In doing so 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) by recognizing the sense of urgency in areas of high medical need.
Regulatory Affairs ensure our products meet the appropriate legislation in order to control their safety and efficacy from early development phases up to the coordination of approval and registration. Regulatory Affairs are the crucial link between the company, the products and the regulatory authorities and they advise on and coordinate 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 in line with the direction of RA Leadership. The candidate is responsible for creating and implementing a global regulatory CMC plan and prepare content plans for regulatory CMC activities to ensure the CMC regulatory strategy is aligned with regulatory requirements and expectations, and that regulatory documents are prepared in high quality and submitted in a timely manner. The associate director closely collaborate with CMC, Manufacturing and Quality and other RA functions to provide direction on the application and interpretation of global CMC regulations (primarily US and Europe) to support the overall project goals. The Associate Director RA-CMC will also closely collaborate the RA Product Lead to align with the 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 ensures alignment with RA LeadershipAssure alignment with the overall global strategy through partnership and communication with other Regulatory FunctionsExecute Regulatory CMC Strategy, activities and deliverables for assigned products in alignment with the overall regulatory strategy and company goalsPlans for program deliverables in cross functional meeting such as regulatory sub-teams, CMC Team meetings and submission meetingsParticipate in the Product team as the Regulatory CMC expert ,working regularly with CMC, Manufacturing and Quality functions to provide regulatory expertise for assigned productsRepresent Regulatory Affairs in cross functional teams and provide regulatory oversight and guidance to project teams on compliance matters independentlyLead and plan the content in the preparation of strategic regulatory documents throughout the product lifecycleLead and participate in regulatory CMC document planning, authoring, review , comments resolution, approval process, and delivery of submissions throughout the product's life cycle including briefing documents, IND/CMC, annual reports and support the preparation of the team for negotiations with FDA, EMA and other health agenciesIdentify 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 plansGuide 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 requirementsConduct Regulatory intelligence and follow evolving 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 pipelineEffectively communicate with internal teams and health authorities where applicable.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 StrategyStrong 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 AuthoritiesIn addition to a RA CMC Background, understanding of pre-clinical/clinical regulatory requirements for drug developmentUnderstanding 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 functionAbility to create, plan and implement a submission processAbility 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 settingsWorks 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.
Location:Lexington, MAEducation level:Master or PhD
Job category:Regulatory AffairsTarget start date:12/1/2024
Work Location:HybridShift:1st
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 bring innovative therapies or unmet medical needs to patients. In doing so 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) by recognizing the sense of urgency in areas of high medical need.
Regulatory Affairs ensure our products meet the appropriate legislation in order to control their safety and efficacy from early development phases up to the coordination of approval and registration. Regulatory Affairs are the crucial link between the company, the products and the regulatory authorities and they advise on and coordinate 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 in line with the direction of RA Leadership. The candidate is responsible for creating and implementing a global regulatory CMC plan and prepare content plans for regulatory CMC activities to ensure the CMC regulatory strategy is aligned with regulatory requirements and expectations, and that regulatory documents are prepared in high quality and submitted in a timely manner. The associate director closely collaborate with CMC, Manufacturing and Quality and other RA functions to provide direction on the application and interpretation of global CMC regulations (primarily US and Europe) to support the overall project goals. The Associate Director RA-CMC will also closely collaborate the RA Product Lead to align with the 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 ensures alignment with RA LeadershipAssure alignment with the overall global strategy through partnership and communication with other Regulatory FunctionsExecute Regulatory CMC Strategy, activities and deliverables for assigned products in alignment with the overall regulatory strategy and company goalsPlans for program deliverables in cross functional meeting such as regulatory sub-teams, CMC Team meetings and submission meetingsParticipate in the Product team as the Regulatory CMC expert ,working regularly with CMC, Manufacturing and Quality functions to provide regulatory expertise for assigned productsRepresent Regulatory Affairs in cross functional teams and provide regulatory oversight and guidance to project teams on compliance matters independentlyLead and plan the content in the preparation of strategic regulatory documents throughout the product lifecycleLead and participate in regulatory CMC document planning, authoring, review , comments resolution, approval process, and delivery of submissions throughout the product's life cycle including briefing documents, IND/CMC, annual reports and support the preparation of the team for negotiations with FDA, EMA and other health agenciesIdentify 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 plansGuide 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 requirementsConduct Regulatory intelligence and follow evolving 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 pipelineEffectively communicate with internal teams and health authorities where applicable.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 StrategyStrong 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 AuthoritiesIn addition to a RA CMC Background, understanding of pre-clinical/clinical regulatory requirements for drug developmentUnderstanding 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 functionAbility to create, plan and implement a submission processAbility 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 settingsWorks 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.