4D Molecular Therapeutics

4D Molecular Therapeutics Emeryville, CA, USA
Sep 17, 2019
Full time
4D Molecular Therapeutics, located in Emeryville, CA is a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. At 4D Molecular Therapeutics, our goal is to design and develop transformative gene therapy products using our proprietary adeno-associated virus variants platform to treat serious unmet medical conditions. 4D is in need of a talented and motivated Associate Director/Director to join our team in supporting our viral drug therapy research efforts as we contribute to the future of gene therapeutics. SUMMARY: This is a key executive position within 4D Molecular Therapeutics (4DMT). Reporting to the Chief Medical Officer, this individual will lead a team of pre-clinical scientists and medical experts bringing innovative gene therapies to patients. This individual will have a high level of passion for 4DMT's mission and the ability to operate as an effective strategic, as well as tactical, thinker. The individual will also be effective as a hands-on manager and producer of individual work. Candidates should be an MD/PhD or an MD or equivalent in relevant disciplines, with significant experience in clinical research and development. You will lead a cross-functional team developing therapeutic candidates from concept, design, through IND filing and clinical Proof of Concept (POC) studies (Phase 1-2 trials). MAJOR RESPONSIBILITIES : Set, communicate and drive strategy for TR&D for therapeutic products in alignment with the broader organization of 4DMT. Prioritize and allocate resources, to meet portfolio needs. Lead the decision-making process with other relevant functional group heads regarding indications, targets and products' progression from concept, design, preclinical development and Phase 1-2 clinical testing. Recruit, convene and manage Scientific and/or Clinical Advisory Boards comprising world-class expertise. Provide input on scientific strategy from the earliest consideration of new targets through development, including risk, scientific challenges, timing of resource investment and development of go/no go decisions. ‪Direct and coordinate the implementation of preclinical study programs for pharmacology studies in support of the lead to candidate optimization process. ‪Design, implement and write-up comprehensive series of pharmacology studies to address preclinical exposure, develop pharmacokinetics and pharmacodynamics profiles and generate GLP toxicology and bio-distribution packages suitable for supporting IND filings. ‪Design, implement and manage externally facing in vivo studies with CROs and academic centers, including drafting proposals, budget oversight and managing deliverables. Provide direct hands-on responsibility for all TR&D interactions as well as grant writing and management with rare disease advocacy groups. Participate on project teams and direct activities to ensure regulatory compliance of pre-clinical and clinical research activities. Support regulatory affairs in their preparation, review and submission of necessary pre-clinical and clinical regulatory information to regulatory agencies worldwide. Define clear human POC study outcome criteria for decision-making on new product candidates. Foster strong external clinical collaborations to maintain state-of-the-art clinical approaches. Together with the clinical operations and project management functions, contribute to the quality of clinical protocols, study reports, investigator brochures and regulatory submission documents. Involvement in all aspects of the design, conduct, monitoring, interpretation, and reporting of clinical trials. Contribute senior level expertise to alliance, business development, and due diligence exercises with external entities and licensing meetings, as well as other critical external interfaces such as public health regulatory bodies. KNOWLEDGE AND SKILLS: MD/PhD, MD or equivalent; experience with translational and clinical R&D. Prior leadership position/s within the pharmaceutical/biotech industry or academic medical centers, with a significant history of research and relevant clinical experience. Reputation for inspiring a culture of continuous improvement, innovation and quality, leading and mentoring at the highest level of achievement. Ability to support and impact experts, scientists and project deliveries across functions in research, development and beyond. Strong written and oral communication skills, with demonstrated ability to present ideas, information and data effectively via one-on-one discussions, team meetings. Excellent time management skills (schedules, timelines, task prioritization) and the ability to adapt to changing priorities. Self-starter who proactively establishes effective working relationships and builds partnerships with other disciplines. Adaptability, flexibility, independence and resourcefulness to roll-up-sleeves and multi-task in order to thrive in a small company environment. Ability to proactively identify challenges, possess strategic foresight and implement appropriate course of action. We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development.
4D Molecular Therapeutics Emeryville, CA, USA
Sep 17, 2019
Full time
4D Molecular Therapeutics, located in Emeryville, CA is a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. At 4D Molecular Therapeutics, our goal is to design and develop transformative gene therapy products using our proprietary adeno-associated virus variants platform to treat serious unmet medical conditions. 4D is in need of a talented and motivated Associate Director/Director to join our team in supporting our viral drug therapy research efforts as we contribute to the future of gene therapeutics. This is an opportunity to join a rapidly growing dynamic platform biotechnology company with multiple products in development. The successful candidate will join a team of outstanding pre-clinical and clinical scientists with the opportunity to gain broad experience in cutting-edge gene therapy, from bench-to-bedside. SUMMARY: The Clinical Scientist/Senior Clinical Scientist will play an integral role in facilitating successful and timely initiation and completion of 4DMTs clinical development programs working with members of Translational Medicine, Clinical Operations, Program Management, Clinical Research Organizations (CROs) and Investigators to plan, execute and monitor medical aspects of selected clinical trials. The successful candidate will operate in a highly detailed and organized fashion with the goal of efficiently driving each program through the clinical development process. MAJOR RESPONSIBILITIES: Collaborate with the translational medicine teams on the design and implementation of clinical studies, the review and interpretation of study results, providing deliverables in approved timeframes. Analyze and review safety and efficacy data to establish the presence or absence of trends and follow up as appropriate. Lead or collaborate in the preparation of clinical study related documents including protocols, charters, statistical summary reports, meeting presentations, publications, and clinical sections of regulatory documents. Engage with investigators, KOLs, SABs, as necessary. Engage with site staff, CROs, and site monitors to maintain enthusiasm and support enrollment for ongoing clinical studies. Review and interpret scientific knowledge of competitor landscape (molecule / indication). Maintain awareness of internal and external developments (scientific, clinical, competitive, and regulatory) including attendance at major scientific conferences. Present clinical strategy and data to internal and external audiences (such as investigator meetings). Provide support for business development activities, such as due diligence and research collaborations. Maintains knowledge of ICH-GCP, external regulations and procedures. KNOWLEDGE AND SKILLS: RN, MS, PhD or PharmD, or equivalent. Advanced degree candidates preferred. Clinical science experience with 2+ years of drug development with experience in ophthalmology, cardiovascular disease, lung or neuroscience, preferred. Expertise in clinical trial design, implementation, regulatory document preparation and statistics. Ability to analyze and interpret clinical and efficacy data and develop written reports and presentations of those data. Able to manage priorities and competing demands in a growing biotechnology company with a broad pipeline of products. Excellent presentation skills for both internal and external audiences. Ability to balance independent work and team interaction. Well-developed interpersonal skills, and ability to build strong positive relationships. Good leadership and management skills to manage internal and external collaborations and work well within cross-disciplinary teams.We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development. Ability to travel, approximately 10%. We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development.
4D Molecular Therapeutics Emeryville, CA, USA
Sep 16, 2019
Full time
4D Molecular Therapeutics, located in Emeryville, CA is a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. At 4D Molecular Therapeutics, our goal is to design and develop transformative gene therapy products using our proprietary adeno-associated virus variants platform to treat serious unmet medical conditions. 4D is in need of a talented and motivated Associate Director/Director to join our team in supporting our viral drug therapy research efforts as we contribute to the future of gene therapeutics. The generation of novel AAV vectors is exciting, and it makes for a very interesting challenge, to advance our knowledge of these brand new vectors. You will have the opportunity to develop and implement both protein- and DNA-based assays for AAV products. You get to work on cutting edge science (with opportunities to present and publish) and to make important contributions to IND filings for novel therapeutics that will enable 4D to advance our therapeutic products into patients. MAJOR RESPONSIBILITIES: Designing and managing cell-based experiments to determine tropism, mechanism of transduction, and other characteristics of novel AAV vectors Designing and managing non-GLP preclinical studies; analyzing and/or confirming data related to non-GLP preclinical studies to determine characteristics of novel AAV vectors Managing preclinical CRO activities Writing and editing preclinical study reports and preclinical sections in support of regulatory submissions Collaborating with cross-functional teams (including CMC, human cell and disease modeling, and translational medicine) to ensure that studies are performed in a quality, timely, and scientific manner Contributing to the management of design, optimization, verification, and execution of current and new assays Demonstrating innovative design, development, and execution of research projects through literature review, expertise, and scientific teamwork Maintaining a current understanding of preclinical and clinical gene therapy literature and methodologies; actively applying new concepts and technologies as appropriate Maintaining compliance to company Environmental Health and Safety policies, procedures, and practices Effectively mentoring junior level staff and acting as a resource for providing scientific expertise Contributing to building a culture that embraces continuous learning, improvement and innovation and encourages team members to expand their technical skill base and deepen their gene therapy expertise. Utilizing appropriate scientific approaches to understand characteristics of adeno-associated virus variants to drive decisions and assess potential application of company's lead adeno-associated virus vectors and products KNOWLEDGE AND SKILLS: Ph.D. degree and at least two years of post-doctoral experience in one of the following disciplines: Bioengineering, Virology, Molecular Biology, Pharmacology, Biochemistry or a related field; D.V.M. or M.D. also acceptable Knowledge and 3-5 years working experience in preclinical research and development (adeno-associated viral vectors preferred) Solid understanding of molecular biology and protein chemistry techniques such as qPCR, qRT-PCR, mammalian cell culture, immunoblot, viral transduction, ELISA, and immunocytochemistry/immunohistochemistry procedures. Proven experience in design, coordination, analysis, and documentation of preclinical studies Successful demonstration of ability to work independently in designing and developing executing new protocols Successful demonstration of ability to rigorously analyze and interpret diverse, multidisciplinary data sets Ability to multi-task and support more than one project simultaneously Ability to work independently and cooperatively in a team-based environment; supportive of multiple viewpoints and approaches Understanding and knowledge of key scientific software programs (including Microsoft Office, JMP or Prism, and Adobe Photoshop) Strong organizational skills, analytical skills, and problem-solving skills Strong communication skills (both written and oral) with demonstrated ability to present ideas, information and data effectively via one on one discussions, team meetings, and external partnership interactions We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development.
4D Molecular Therapeutics Emeryville, CA, USA
Aug 30, 2019
Full time
4DMT is at the forefront of cutting-edge science and technology with proprietary AAV vectors that deliver transgene payloads to the right place for patients. Your creativity and innovative design, development and execution expertise in purification and formulation development will be critical to helping us take 4DMT to the next level. Your work will directly impact both Process Development and Manufacturing. This role will require close collaboration with the Analytical Sciences & Bioinformatics (ASB) team. This is an opportunity to join a rapidly growing pre-IPO company as a key member of the process development team. You will be involved in the build-out and outsourcing decisions for a new, expanded process development laboratory, and have the opportunity to work on many products across multiple therapeutic areas. The diverse nature of our AAV vectors makes this position ideal for scientists looking to solve challenging puzzles, with much work to be done to advance the field to help accelerate the delivery of high value therapeutics to patients. Moreover, you will have opportunities to present and publish as well as to make important contributions to regulatory filings for novel therapeutics. MAJOR RESPONSIBILITIES: Leverages strong understanding of biophysical characterization techniques (including DLS, DSC, CE and HPLC-based methods, UV, and AUC) for pre-formulation studies. Designs, executes and interprets experimental data. Responsible for management of outsourced formulation studies, working with CROs. Develops formulations based on physiochemical information derived from pre-formulation studies to support the needs for drug delivery. Works closely with project leads to define deliverables and project timelines. Responsible for IND-enabling characterization of therapeutic and reporter viral transgenes, using a variety of methodologies to test for product stability and comparability. Work with Process development team to develop and scale-up multi-step purification process for the production of AAV variants. Applies knowledge derived from pre-formulation studies to aid in appropriate buffer and excipient selection. Authors SOPs and technical reports, presents experimental data in project and group meetings. Demonstrates innovative design, development and execution of projects through literature review and scientific teamwork. Reviews data and supports troubleshooting issues that occur during development, scale-up, and manufacturing. Contributes to building a culture that embraces continuous learning, improvement and innovation, and encourages team members to expand their technical skill base and deepen their gene therapy expertise. Maintains a current awareness and contributes to current scientific literature; actively applies new concepts and technologies as appropriate. Leads and supports research initiatives such as new technology development and continuous improvement projects KNOWLEDGE AND SKILLS: A PhD in Biochemistry, Chemical Engineering, or related scientific field is preferred. A minimum of 2 years' work experience in drug product development is required. Exceptional MS or BS candidates with demonstrated capabilities in process development and formulation will be considered (e.q. MS with >6 years, B.S. with >10 years) Must have a solid understanding of formulation development for biologics (AAV preferred), protein chemistry, and experience with standard analytical characterization techniques (DLS, DSC, IEC, HPLC, particle characterization) Working knowledge in design of experiments (DoE) and associated software Skilled in the art of protein purification techniques including column chromatography, filtration, and TFF Working knowledge of AKTA chromatography system with Unicorn Software or alternate system Experience working with external vendors and CROs is desirable Successful demonstrated ability to work independently and lead others in designing and developing experiments, analyzing and interpreting data in a rigorous way Ability to multi-task and support more than one project simultaneously Ability to work in a team-based environment, cooperatively and supportive of multiple viewpoints and approaches Strong organizational skills, analytical and problem-solving skills Strong written and oral communication skills, with demonstrated ability to present ideas and information and data effectively via one-on-one discussions, team meetings and partnership interactions 4D Molecular Therapeutics, located in Emeryville, CA is a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. At 4D Molecular Therapeutics, our goal is to design and develop transformative gene therapy products using our proprietary adeno-associated virus variants platform to treat serious unmet medical conditions. 4D is in need of a talented and motivated Associate Director/Director to join our team in supporting our viral drug therapy research efforts as we contribute to the future of gene therapeutics. This is an opportunity to join a rapidly growing dynamic platform biotechnology company with multiple products in development. The successful candidate will join a team of outstanding pre-clinical and clinical scientists with the opportunity to gain broad experience in cutting-edge gene therapy, from bench-to-bedside. We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development.
4D Molecular Therapeutics Emeryville, CA, USA
Aug 30, 2019
Full time
4D Molecular Therapeutics (4DMT) , a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development (Emeryville, CA) has a Research Associate/Senior Research Associate opportunity in its Analytical Sciences and Bioinformatics team. 4DMT is at the forefront of cutting-edge science and technology with proprietary AAV vectors that deliver transgene payloads to the right place for patients. This is an opportunity to join a rapidly growing pre-IPO company as a key member of the ASB team. Your talents, excellent attention to detail, and insatiable desire to learn will be critical to the success of our team and in achieving 4DMT's vision. You will execute routine testing in support of process development and manufacturing and contribute to the development of analytical assays for characterization of therapeutic products. In addition, you will need to make experimental observations, review and analyze data for presentations and reports, and share maintenance responsibilities such as reagent preparation, material ordering, and general lab upkeep. Responsibilities: Follow proper safety precautions and laboratory technique in the use of chemical compounds and reagents Perform established analytical methods such as PCR (qPCR/ddPCR), Electrophoresis, and ELISA Design, execute and report qualification assays under the supervision of senior group members Write and revise documents such as SOPs and technical reports Analyze data, identify trends and troubleshoot assays Provide instrumentation care, maintenance, and troubleshooting Perform general laboratory duties such as stocking and ordering supplies Perform other duties as assigned Knowledge & Skills: Bachelor's degree in a Life Science discipline 0 - 3 years industry experience PCR knowledge/experience required Proficient in Microsoft Word, Excel, Power Point and other applications Ability to communicate and work in a team environment Ability to think critically and demonstrate problem-solving skills Knowledge of cGMP documentation is preferred Knowledge: The successful candidate must have some background in protein chemistry and molecular biology techniques Working knowledge of PCR/qPCR/ddPCR, gel electrophoresis, and ELISA is preferred Excellent communication skills are a must for this position, both verbal and written Basic understanding of ICH guidelines Good time management and the ability to multitask in a fast-paced environment is a must We are 4D Molecular Therapeutics (4DMT) -- a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic diseases. Our goal is to design and develop transformative gene therapy products using our proprietary adeno-associated virus variants platform to treat serious unmet medical conditions. While AAV gene therapy pioneers are making great strides with the first wave of products, hurdles still exist with the use of 1st & 2nd generation vectors (naturally occurring primitive viral capsids). 4DMT's next-generation Therapeutic Vector Evolution platform generates targeted, optimized and proprietary AAV vectors that have the potential to overcome the delivery and immunological challenges currently facing the field, ultimately unlocking the full potential of gene therapy. We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure severe genetic and rare diseases with high unmet medical need. We seek the best people in the industry who identify with our mission and innovative results-driven approach. We offer an inspiring and collaborative environment that encourages individual contributions, recognizes performance in multiple ways, offers uniquely generous benefits and provides challenging opportunities for career development.