Chief Scientific Officer

Genethon is seeking a visionary leader and respected scientist, with expertise in gene-based therapies and a track record of translating science into medicines, to join the team as Chief Scientific Officer (CSO). The ideal candidate will have the following mix of personal and professional characteristics:

• PhD in a relevant discipline.
• Extensive experience working in discovery, preclinical development, and/or translational medicine, with multiple years of R&D management experience.
• Substantial knowledge of gene therapy and the key challenges associated with this space.
• Strong leadership skills and excellent interpersonal skills, with a track record of building high-performing teams.

OVERVIEW

Genethon, a non-profit research and development laboratory established by AFM-Telethon in 1990, was a pioneer in genomics, leading to the first human genome map, and has since become a trailblazer in the discovery and development of gene therapies for rare diseases. On a mission to change the lives of patients suffering from rare genetic disorders, Genethon is a fully integrated organization, comparable in its structure to a biotech; with over 220 scientists and experts, its R&D teams span from translational science and CMC to clinical trials. Genethon’s R&D has been at the origin, or has contributed to, a large number of therapies currently in clinical trial, in advanced preclinical phase, and to one product currently on the market. These therapies are either developed in the framework of high-profile pharma/biotech partnerships, or by Genethon on its own. In particular, Genethon’s R&D is at the origin of seminal preclinical work and IP for Zolgensma® (commercialized by Novartis), has been at the origin AT132 (resamirigene bilparvovec, licensed to and developed by Audentes/Astellas Gene Therapies), and has contributed to Lumevoq ® (licensed to Gensight Therapeutics), amongst multiple other gene therapies targeting neuromuscular pathologies, immune system diseases, ocular and metabolic diseases. In addition, scientists at Genethon have been at the origin of approx. 1000 patent applications and1500 peer reviewed articles.

Genethon evolves in one of EU's most stimulating environments for gene therapy R&D and ison the same campus as EU’s largest GMP manufacturing site for gene therapy, Yposkesi, originally a spin off from Genethon. Genethon is located within Genopole, the largest biotech hub in France, also host to ISTEM, a major EU site for stem cell therapy R&D. Genethon entertains close connections with APHP (Paris University Hospital’s), INSERM, and has academic and industry collaborations worldwide. Genethon also enjoys particularly close ties with the associative world, through the worldwide network forged by AFM-Telethon, the largest rare-diseases patient organization in Europe.

THE POSITION

Genethon seeks a visionary leader and respected scientist, with expertise in gene-based therapies and a track record of translating science into medicines, to join the team as Chief Scientific Officer (CSO). Motivated by a sense of urgency and known for their success and integrity in prior endeavors, the CSO will oversee responsibility for driving and delivering on Genethon’s highly focused mission. Reporting to the Chief Executive Officer, this person will build and lead scientific strategy for the entire organization, developing long-term research plans, keeping Genethon at the forefront of innovation for gene therapy.

Key responsibilities include:

• Identify new research opportunities for therapies, indications, and gene therapy-related technologies (vectors, gene editing), and, depending on the candidate’s profile, manufacturing technologies.
• Contribute to and drive the development and execution of the overall R&D strategy, advising on translational and clinical research plans to advance novel therapies.
• Identify areas of unmet need in research and medicine and develop innovative research programs to address these needs.
• Develop and maintain strategic partnerships with investigators, researchers, industry stakeholders, regulators (EU and US), and other government agencies to advance Genethon’s scientific agenda and programs.
• Maintain connections with the scientific community, keeping abreast of the latest scientific discoveries and industry trends.
• Oversee all aspects of Genethon’s scientific endeavors, including development of new products and ongoing research, to ensure that quality standards are met.
• Evaluate scientific data and provide scientific guidance to inform investment decisions, research programs, and grants.
• Ensure appropriate scientific review, study design, feasibility, regulatory guidance, project management, timelines, and budgets.
• Develop, maintain, and comply with the organization’s research policies and procedures, all applicable laws and regulations, and adhere with codes of ethical principles and standards of professional conduct.
• Organize necessary stakeholders for internal meetings, advisory groups, and roundtables to inform overall scientific strategy and advance critical R&D questions.
• Develop scientific communications plan to support all research initiatives, including but not limited to: written publications, submissions to scientific meetings, oral presentations, audits, and due-diligences.
• Collaborate with internal cross-functional teams and external partners and consultants to ensure alignment and efficient execution of research and development plans.
• Mentor and develop leaders at all levels and maintain strong, collaborative relationships across the organization.
• Create and advise on scientific content for new funding and technological opportunities.
• Participate in business development efforts, aiding leadership in decisions regarding asset in/out-licensing and/or collaborations.
• Collaborate with all research institutes created by AFM Telethon, in particular Istem (cell therapy) and Institute of Myology.

The ideal candidate will have the following mix of personal and professional characteristics:

• PhD in a relevant discipline.
• Extensive experience working in discovery, preclinical development, and/or translational medicine, with multiple years of R&D management experience.
• Substantial knowledge of gene therapy and the key challenges associated with this space.
• The ability to contribute technically to multiple programs using mainly AAV vector technologies and build a strong vision for leveraging next generation of gene therapy technologies.
• Extensive experience presenting scientific and clinical information to a range of audiences.
• The adaptability and flexibility to both lead a big-vision strategy and be hands-on to drive the execution of projects in a growing, fast-paced, dynamic environment.
• Experience interacting with government agencies and collaborating with academic and industry partners.
• Demonstrated success in establishing and maintaining relationships with key opinion leaders and other external partners across a variety of disciplines.
• Strong leadership skills and excellent interpersonal skills, with a track record of building high-performing teams.
• High emotional intelligence to enable favorable group dynamics and the ability to influence others at all levels.
• Outstanding written and verbal communication skills.