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Scientist, AAV & Gene Editing Engineering

Employer
4D Molecular Therapeutics
Location
Emeryville, CA, United States
Start date
Dec 15, 2020

View more

Discipline
Clinical, Medical Writing, Science/R&D, Biotechnology, Genetics
Required Education
Doctorate/PHD/MD
Position Type
Full time
Hotbed
Biotech Bay

Job Details

4D Molecular Therapeutics (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.

4DMT is focused on attracting and retaining 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. Join us in creating the ultimate gene therapy products to cure genetic diseases in children and adults. 4D Molecular Therapeutics, located in Emeryville, CA.

GENERAL SUMMARY:

The 4DMT process development team is looking for a talented scientist with experience in the design and implementation of gene and genome editing towards improving cell-based AAV production. The role allows for extensive creativity, innovation and opportunity to develop novel processes, allowing for extensive coordination within and across teams. You will work closely with your peers in downstream process development, the analytical team and colleagues in research. The group works closely with our counterparts in both manufacturing and research; exchanging scientific ideas, platform improvements and AAV expertise will be a frequent and encouraged activity.

RESPONSIBILITIES:
  • Lab & Experimental Activities: 65% of time
  • Design studies that implement gene circuit design, homology-directed repair, single nucleotide polymorphisms, viral transgenesis and clonal selection/expansion to direct and control cell function
  • Develop studies that mature process knowledge towards AAV structure/function
  • and improve AAV production yields/product quality and other quality attributes
  • Implement strategies to produce engineered cells that modulate AAV production and function
  • Execute gene and genome edits using viral and non-viral methods, including transient transfection and electroporation
  • Optimize transfection conditions: multiplexing engineered cell lines with improvements in media formulation, transfection adjuvants, cell and other critical process parameters
  • Develop seed train, transfection and harvest processes with considerations towards reducing cost of goods and manufacturing
  • Draft study designs, standard operating procedures and maintain an electronic lab notebook


  • Cross-functional & communication : 35% of time
  • Collaborate with process & analytical teams to screen and evaluate drug candidates across a range of indications
  • Review and present data cross-functionally at department meetings and larger forums
  • Maintain strong working relationships with external collaborators and/or CDMOs
  • Contribute to a culture that embraces continuous learning, improvement and innovation
  • Draft technical reports, contribute to IND sections and other regulatory documents
  • Maintains current awareness and contributes to current scientific literature; actively applies new concepts and technologies


QUALIFICATIONS:
  • Education:
  • PhD in molecular biology, cell biology virology, genetics or related field, or
  • Master in molecular biology, cell biology, virology, genetics or related field
  • Experience:
  • PhD with 0-3 years (or Master's with 5+ years) relevant experience
  • Skills:
  • Knowledgeable and hands-on experience with nucleic sequence design, cloning and cell-incorporation
  • Experience modulating immortalized cell lines using methods that align with cGMP manufacturing
  • Strong problem-solving, analytical skills and creativity
  • Experimental study design, leveraging past studies and extracting from the scientific community
  • Strong written and oral communication skills with experience presenting scientific information
  • Excellent time management organizational skills, with ability to handle multiple tasks simultaneously in a high-growth, fast-paced environment


  • Work Location:
  • This position requires the ability to work on location in the 4DMT lab at least 3-4 times a week. There may be some remote work during Shelter in Place.
  • Once Shelter in Place has been lifted this will be full-time position at the 4DMT Emeryville location


4DMT provides equal employment opportunities to all employees and applicants for employment without regard to race, color, religion, gender, sexual orientation, national origin, age, disability, genetic information, marital status, status as a covered veteran and any other category protected under applicable federal, state, provincial and local laws.

Equal Opportunity Employer/Protected Veterans/Individuals with Disabilities

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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