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Associate Director/Director, Preclinical Vector Characterization

Employer
4D Molecular Therapeutics
Location
Emeryville, CA
Start date
Oct 30, 2019

View more

Discipline
Science/R&D, Molecular Biology, Pharmacology, Virology
Required Education
Doctorate/PHD/MD
Position Type
Full time

Job Details

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

 

Company

4DMT is a gene therapy company with a transformative discovery platform --Therapeutic Vector Evolution --that enables our “disease first” approach to product discovery and development, thereby allowing us to customize our AAV vectors to target specific tissue types associated with the underlying disease.


At 4DMT we are creating and developing optimized “biopharmaceutical grade” AAV vectors that will allow us uniquely to unlock the full potential of gene therapy. Our customized and proprietary AAV vectors are each designed, according to a 4DMT Target Vector Profile, to deliver genes to specific cells and tissues in the body to eradicate both rare and large market diseases.


While AAV gene therapy pioneers are making great strides with the first wave of products, hurdles still exist with the use of conventional vectors. The most commonly used AAV capsids (e.g., AAV2, 5, 8 and 9) were identified either 1) as contaminants in lab stocks of adenovirus, or 2) through monkey tissue processing. Therefore, these conventional vectors are not targeted specifically to any tissue in the body. This can lead to inefficient and non-specific delivery, thus requiring extremely high doses and potentially resulting in toxicities (including inflammation), high manufacturing burdens and suboptimal efficacy. In addition, suboptimal routes of delivery can be required for delivery to the right tissues (e.g., subretinal delivery to the retina). Finally, pre-existing neutralizing antibodies in many patients can limit efficacy, clinical trial enrollment and market sizes. 4DMT Innovation empowers us to create new and improved next generation AAV capsids to allow targeted delivery of our products by the optimal clinical route of administration.


At 4DMT we use our robust discovery platform, termed “Therapeutic Vector Evolution,” to create customized and proprietary gene delivery vehicles (novel AAV vectors) to deliver genes to specific tissues and cells in the body by the optimal route of administration. We use the power of evolution to create and refine these optimized vectors through genetic diversification and then natural selection in vivo.


4D Purpose Statement:  Boldly Innovating to Unlock the Full Potential of Gene Therapy for Countless Patients


GUIDING PRINCIPLES

 

  • Dare to Cure - Patients are waiting. Create big dreams for patients and take calculated risks to achieve them
  • Break Boundaries - Question the status quo and innovate beyond conventional approaches
  • Beyond Yourself - It's about patients, their families, our team, our families. We strengthen each other.
  • Prepare and Execute Relentlessly - Start with the end in mind, over-prepare and make contingency plans to your contingency plans


We invite all of you who share our passion and vision in creating the ultimate gene therapy products to cure genetic and rare diseases in children and adults. 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 generous benefits and provides challenging opportunities for career development. 
 

Company info
Website
Phone
510-505-2680
Location
5858 Horton St. Suite 455
Emeryville
California
94608
United States

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