Featured lncRNA Job – Head of Translational Development

RaNA Therapeutics, located in Cambridge, Massachusetts, is pioneering the discovery and development of a new class of RNA-based therapeutics that selectively activate protein expression. We focus on developing treatments for diseases with a significant unmet need that are difficult or impossible to address with existing technologies. Our lead programs include the upregulation of SMN2 for the treatment of Spinal Muscular Atrophy and the upregulation of Frataxin for the treatment of Friedreich’s Ataxia. Along with these lead programs, we are targeting other rare genetic diseases, hepatic diseases, and inflammatory diseases. We are dedicated to translating cutting-edge science in the areas of gene regulation, lncRNA and epigenetics into drugs that will have a significant impact in the treatment of life-threatening diseases.

We are seeking a highly motivated and scientifically rigorous individual to join RaNA and lead our Translation Development efforts.  The successful candidate will report to the Chief Scientific Officer and be an integral part of the Research and Development Leadership team.

Summary of Role

The Head of Translational Development will be highly analytical, decisive, goal-oriented, and timeline sensitive while maintaining the highest of scientific and ethical standards. S/he must be capable of representing the Company externally in all technical matters, collaborating with other R&D leadership and external alliance partners or collaborators and assisting in the identification of new business initiatives and in-licensing opportunities. The successful candidate will be responsible for design and implementation of Translational Development strategy, providing support and guidance to early preclinical and clinical programs through registration and commercialization.  In addition, s/he will effectively lead and manage the Translational Development department, comprised of Pharmacology, Pharmacokinetics and Drug Metabolism, Toxicology, Bioanalysis and Biomarkers, ensuring that resources are applied appropriately, that Translational Development functions have the leadership, experience and expertise needed to deliver on their goals and that the internal versus external resource balance is maximized. The Head of Translational Development will also establish and develop a broad network of external collaborators in order to maintain cutting edge core competencies, facilitate a proactive outsourcing strategy, and identify novel preclinical development strategies and approaches and to keep abreast of the regulatory environment.

Key Competencies

  • A broad understanding of the drug development process (both large & small molecule) and the ability to provide expert guidance in designing and implementing comprehensive translational development programs for biopharmaceuticals including protein therapies, antibodies, oligonucleotides and small-molecule therapeutics.
  • Demonstrate and maintain comprehensive cross-functional and industry perspective, judgement and ability to apply understanding to individual projects and programs.
  • Vision and ability to build and lead a multidisciplinary scientific team in establishing and executing optimal pre/nonclinical development strategy to deliver on RaNA’s strategic Research and Development and portfolio objectives.
  • Effective integration of activities of Translational Development functions (Pharmacology, Pharmacokinetics and Drug Metabolism, Toxicology, Bioanalysis and Biomarkers) with other key functions within RaNA (e.g. Discovery, Chemistry, Clinical, Regulatory).
  • Ability to effectively represent the Company to key external stakeholders and effectively engage and present to staff at all levels, including senior management, alliance partners and global regulatory agencies, clearly and at the appropriate level of detail in representing Translational strategy, results and findings.
  • Ability to rapidly assimilate/synthesize data and information from diverse sources, make judgments on complex/abstract problems and develop innovative solutions as necessary.
  • Demonstrated ability in problem solving as applied to drug development and program/functional management, including crisp decision-making following appropriate consultation, even in times of ambiguity.
  • Excellent interpersonal skills including active listening, effective meeting management, negotiation, and optimizing group dynamics.  Must be open to the ideas of others and utilize different perspectives to drive towards solutions. Ability to influence through effective communication, respectful challenge, and persuasion across the Research and development organization is essential.
  • Develop and implement translational and early development strategies optimized for therapeutic modality, disease indication, clinical and regulatory development pathways, including development and execution of strategy for quantitative analysis of compounds, immunogenicity assessment and identification and implementation of clinically relevant biomarkers during preclinical and clinical development.
  • Ensure that pre/nonclinical data packages are generated to support robust, rapid development candidate selection, and provide ongoing translational support of these candidates into and through clinical development, registration and commercialization.
  • Establish and maintain high quality systems and procedures for outsourcing, managing and reporting for RaNA portfolio programs and to oversee the generation of documentation for the pre/nonclinical components of regulatory submissions. Identifying and introducing new enabling technologies and identification of external capabilities as necessary.
  • Ability to establish, develop and manage relationships with key external knowledge and opinion leaders (KOLs), alliance partners, collaborators and other lop world class scientific experts.
  • Serve as a member of the RaNA Research and Development Leadership Team, influence the operations and future strategy of the division, fostering a culture of openness, creativity and innovation and personify a sense of urgency and can do spirit.


  • PhD and/or M.D. in Pharmacology, Pharmacokinetics, Toxicology, Biochemistry or related field
  • 12+ years of experience working within the biotechnology or pharmaceutical industry, including previous work in the preclinical evaluation and development of nucleic acid and small molecule therapeutics. Experience in rare diseases and with oligonucleotide-based therapeutics is preferred.
  • Significant experience leading experienced teams of scientists from multiple disciplines.
  • Demonstrated knowledge of global regulatory requirements and expectations for nonclinical packages to support first in human, clinical development, and product registration, including thorough knowledge of GLP, GCP, ICH and other applicable global guidelines.
  • Experience representing function to global regulatory agencies in support of submissions at all stages of development, ie, IND/CTA, CTD, IMPD, MAA/NDA/BLA/JNDA.
  • Extensive experience in the outsourcing and external oversight of GxP compliant translational and pre/nonclinical development studies conducted by qualified suppliers.
  • Evidence of thought/opinion leadership within the industry, such as through peer-reviewed publications or as a participant on expert panels or working groups, is preferred.
  • Must be motivated, creative, energetic, resourceful and adaptable, responding constructively to challenging new ideas, information, situations or criticism.
  • Fluency in written and spoken English is required.
  • Domestic and occasional international travel required.

To apply, please email your CV to careers@ranarx.com.

RaNA Therapeutics is committed to equal employment opportunity. All applicants must have authorization to work in the U.S. RaNA has a competitive employee benefits package that includes health/dental insurance, 401K retirement plan and company match, paid vacation, and much more!

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