Executive Director, Clinical Research -Gene Therapy | BioNJ Talent Services

Executive Director, Clinical Research -Gene Therapy

Contact Information
Contact Name: 
Amicus Therapeutics
Organization Name: 
Amicus Therapeutics
Position Location: 
Cranbury
Company Profile: 

Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-centric biotechnology company focused on discovering, developing and delivering high-quality medicines for people living with rare metabolic diseases.

The cornerstone of the Amicus portfolio is GalafoldTM (migalastat). The lead biologics program in the Amicus pipeline is AT-GAA, an investigational therapy for Pompe disease. Amicus is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases.

Job Description:

Summary of Position

The Executive Director, Clinical Research – Gene Therapy, is responsible for leading the generation of the Clinical Development Plans and the clinical trial protocols that will lead to successful submissions of novel Gene Therapy treatments. In this area of Gene Therapy treatments, the Executive Director, Clinical Research will provide insight to the CMO and the VP, Clinical Research and will collaborate with other functions in the Development Organization to optimize the strategy of Amicus Therapeutics and to implement, execute, analyze and report the clinical studies into global submissions. The overall clinical development strategy for Pompe and Fabry diseases remains with their Clinical Research lead who will collaborate with the Executive Director, Clinical Research – Gene Therapy in the case of Gene Therapies for Pompe and Fabry.

Roles and Responsibilities

The Executive Director, Clinical Research – Gene Therapy (GT) is responsible for:
Leading the clinical development strategy and the Clinical Development Plan (CDP) preparation for the GT clinical programs, and overseeing the generation of the GT clinical trial protocols.
Executing the development plan for the GT clinical programs from early phase studies up to the global submissions and beyond.
Playing a significant leadership role, as well as providing hands on tactical work, in project teams and working in collaboration with relevant team members and peers to ensure the highest level of execution for the GT clinical program.
Working with Clinical Trial Investigators and clinical research organizations, to collaborate on trial site selection, training and ongoing conduct of the study.
Ensuring that studies are performed with the highest quality. Performing data review and analysis, presentations (oral and written) of clinical data, authoring and editing of clinical study reports.
Successfully interact with experts and other external partners in a manner consistent with Amicus’ beliefs.
Participating in preparation of regulatory documents (e.g. Investigator Brochures, CSRs, Briefing documents) and in face-to-face meetings with the regulatory agencies.
Maintaining highest level of medical science and clinical development expertise in Amicus’ fields of interest.
Collaborating on business development activities in support of GT
Perform all responsibilities in compliance with company policies, SOP and guidelines.
Sharing, within the Development Organization and the company, his/her knowledge and expertise in GT to broaden and update the current level of awareness and practice in GT.

Qualifications: 

Qualifications and Background Requirements
Medical Doctor’s (MD) degree and post residency training is required
Completion of a residency in either pediatrics or internal medicine and, preferably, a fellowship in a related subspecialties area (e.g. neurology, genetics).
Significant experience practicing medicine is a plus
OR Ph.D with exceptional background in drug development and gene therapy
 A minimum experience of 10 years in industry is required of which at least 5 years have been spent working on or leading GT projects.
Experience with rare and/or orphan diseases is highly desired. Knowledge of lysosomal storage disorders a significant plus.
Experience in drug development in pediatric populations is a significant plus.
A drug development professional with experience derived from a career in a major pharmaceutical company, or in a biotechnology or biopharmaceutical company environment, including, preferably, a key role in the submission of several successful NDA/BLA and/or MAA.
Experience in pharmacovigilance processes, including development Risk Management Plans, required by global regulatory authorities in the conduct of GT studies and patient follow-up post-GT.
Experience on teams with early and late stage development products.
Strong project management skills, business savvy and business development experience is desirable.
Flexibility and adaptability, able to thrive successfully in small biotechnology company environment.
Excellent oral and written communication skills.
Strong interpersonal skills, a collaborator/ team player, open minded to the diverse opinions of others.
Ability to produce highest quality and timely clinical development work product/ documents with limited direction.
Hands-on performer, ensuring strong execution of clinical trials and other drug development activities.
He or she must also possess a strong sense of personal and professional ethics.
A good balance of confidence and humility.Must be passionate about developing treatments for patients with rare/orphan diseases.

Travel
Travel both domestic and international ± 25% of time

Additional Job or Internship Information
Position Type: 
Full time job
Function: 
Clinical Research