The position will be based either in Princeton, N.J. or Los Angeles, CA
Job Reports To
VP of Biology, Los Angeles, CA
CHDI oversees a portfolio of projects aimed at understanding the natural progression of Huntington’s disease (HD), and identifying impactful therapeutic strategies to pursue with the goal of accelerating bringing therapies to patients. CHDI is focusing on a number of prioritized biological areas including Huntingtin lowering, Huntingtin structure and function, circuitry modulation and human genetics, as entry points for drug discovery and development.
Advancement of our goals require efficient and effective application of state-of-the art technologies across a wide range of platforms. We seek a knowledgeable and accomplished expert in molecular biology and applied research to lead our research efforts involving viral vector development and utilization. A priority focus is deployment of state-of-the-art applications of adeno-associated viral (AAV) vectors as delivery agents to express proteins, RNAi, and guide RNAs in the preclinical research and to interface with our collaborative partnerships in human gene therapy approaches for HD.
The position offers involvement in an innovative model of non-profit rare disease drug discovery and development and participation in an entrepreneurial and collegial work environment with opportunities and resources to contribute significantly to the understanding and treatment of HD.
Job Role Description:
- Accountable for advancing CHDI’s use of AAV (adeno-associated virus) gene delivery in support of HD therapeutics by contributing innovation in vector engineering and optimization, manufacturing and dosing.
- Contribute to the evaluation, prioritization, and implementation of viral gene delivery systems to the CNS, including a current focus on adeno-associated virus (AAV) vectors to investigate the potential for genome editing and gene regulation approaches in support of HD drug development and as a potential HD gene therapy. Strategies include central and systemic administration of novel AAV capsids.
- Contribute to a strategic framework to bring forward drug discovery candidates from internal and external research programs and specifically be accountable for the design, development and utilization of viral delivery vectors (AAV, lentiviral, etc.) and associated “payloads” (encoded proteins, RNAi, guide RNAs) to gain insights into the biology of HD pathophysiology and to support the identification and validation of targets for novel therapeutic development.
- Accountable for assessing and implementing viral based delivery technologies for in vitro cell culture and in vivo rodent and large animal models, as part of a larger team of scientists for in vivo assessment of therapeutic efficacy in suitable animal models of HD.
- Be part of a team overseeing CHDI’s efforts in the molecular and gene therapy clinical development programs targeting HTT expression, interacting with external companies.
- Accountable to establish and manage a collaborative network of contract research and academic scientists engaged in advancing and applying viral delivery vectors and gene modulation technologies.
- Participate in internal and external scientific reviews of biology and technology platforms that would support and advance HD research
- Execute all the necessary process, legal, and business requirements to enable scientific efforts and goals in areas of accountability
- Communicate the state-of-the art opportunities in viral vectors and gene delivery and their relevance and impact to accelerating an understanding of HD biology and candidates for treatments to internal and external parties.
The candidate of choice will have:
- An M.D. or D. in life sciences and relevant postdoctoral training
- A minimum of 5 years of experience as an independent scientist
- Experience in gene therapy and the use of viral delivery vectors in drug discovery, with a particular emphasis in CNS therapeutics.
- Expertise and knowledge in molecular biology and significant experience with viral vector technologies in cell culture and in vivo applications
- Experience in viral production and purification including AAV and lentiviral vectors
- Expertise and experience in the design and characterization of viral delivery to cells and in animal models (rodent and large animal) for target validation and exploration of biological mechanisms
- Significant experience in planning and conducting in vitro, in vivo, and ex vivo genetic and pharmacologic perturbation studies, optimally with experience in the central nervous system
- Demonstrated ability to identify, design, and drive innovative programs and demonstrated evidence of scientific “problem solving” specifically as applied to drug discovery
- Strong analytical skills, critical reasoning, and scientific rigor
- A track record of scientific accomplishment as evidenced by publications in refereed journals and/or securing of research funding support
- Excellent written and oral communication skills, and effective interpersonal skills necessary for the coordination of a large network of external global collaborators
- Evidence of being highly self-motivated and able to work independently
- Able to travel to the various CHDI offices as well globally to interact with collaborators
An ideal candidate might also have:
- Experience with gene therapy studies in humans
- Experience working on Huntington’s disease and/or other neurodegenerative diseases
- Experience in the biopharmaceutical sector
- Productive history of managing external collaborators and collaborations
- Experience in neuropharmacology
- Experience in (stem) cell based assays
- Familiarity with contemporary ‘omics technology platforms and data analyses
Send CV’s to firstname.lastname@example.org