4D Molecular Therapeutics
5858 Horton St. Suite 455
About 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.
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
- 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.
23 articles with 4D Molecular Therapeutics
4D Molecular Therapeutics Appoints John F. Milligan, Ph.D., as Executive Chairman of Board of Directors
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene therapy based on directed evolution, announced the appointment of John F. Milligan, Ph.D. as Executive Chairman of the Board of Directors.
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced the appointment of Susannah Gray to the Board of Directors.
4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of 4D-125 by Intravitreal Injection for the Treatment of X-Linked Retinitis Pigmentosa
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-125 for X-Linked Retinitis Pigmentosa, a blinding and currently untreatable inherited retinal disease.
4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the U.S. Food and Drug Administration has granted Fast Track designation to 4D-310 for Fabry disease, a debilitating lysosomal storage disorder caused by a deficiency of alpha-galactosidase A enzyme activity.
4D Molecular Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of 4D-110 by Intravitreal Injection for the Treatment of Choroideremia
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1 clinical trial of 4D-110, a Roche-licensed product candidate, delivered by a single intravitreal injection for Choroideremia.
4D Molecular Therapeutics, a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced a new agreement with Cystic Fibrosis Foundation to develop precision gene medicines for cystic fibrosis.
Proceeds will be used to advance 4DMT’s three precision-guided AAV gene therapy candidates through initial clinical proof-of-concept, to advance pre-clinical product pipeline and proprietary next-generation Therapeutic Vector Evolution platform, and to expand internal GMP manufacturing capabilities – 4D-310 for the treatment of Fabry Disease, 4D-125 for the treatment of XLRP and 4D-110 for the treatment of choroideremia are each expected to enter the
Before the end of this year, 4DMT intends to launch clinical trials for 4D-310, its product candidate for the treatment of Fabry disease, and 4D-125 and 4D-110, its two ophthalmology product candidates.
Check out this article for the impacts that Biotech Bay companies are experiencing due to COVID-19.
4D Molecular Therapeutics Announces Upcoming Oral and Poster Presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy
Oral presentations to include data on 4D-310 Product Candidate for Fabry Disease and 4D-A101 Aerosol Vector for Cystic Fibrosis and other Lung Diseases
4D Molecular Therapeutics Announces Presentation of Preclinical Data at the 6th International Update on Fabry Disease and Provides Clinical Update
Novel vector, 4D-C102, demonstrates superior performance compared to commonly used AAV1, AAV8, and AAV9
2/22/2019Companies make leadership changes to executive teams, including additions at Allergan, Surmodics, Arix, RedHill, Cerevel, Enzyvant, and more.
4D Molecular Therapeutics Expands Leadership Team with Appointments and Promotions of Key Executives
4D Molecular Therapeutics, a world-leader in Therapeutic Vector Evolution for adeno-associated virus gene therapy vector discovery and product development, announced the appointments of Fred Kamal, PhD, as chief technical officer, and August Moretti as chief financial officer.
4D Molecular Therapeutics Announces Collaboration to Advance Gene Therapies for Retinal Diseases with Oregon Health & Science University’s Casey Eye Institute and Oregon National Primate Research Center
4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, today announced a research and development collaboration with the Casey Eye Institute (CEI) and the Oregon National Primate Research Center (ONPRC) at Oregon Health & Science University (OHSU).
4D Molecular Therapeutics Announces FDA Orphan Drug Designation Granted to 4D-110 for the Treatment of Choroideremia
4D Molecular Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to 4D-110 for the treatment of choroideremia.
4D Molecular Therapeutics snagged $90 million in a Series B financing round. The company intends to use the funds to advance its Therapeutic Vector Evolution platform and pipeline of next-generation AAV gene therapeutics.
AstraZeneca’s MedImmune and 4D Molecular Therapeutics forged a collaboration to develop a gene therapy for chronic lung disease.
4D Molecular Therapeutics and MedImmune Collaborate to Design, Develop and Commercialize AAV Gene Therapy for Chronic Lung Disease
MedImmune and 4D Molecular Therapeutics announced a collaboration to develop and commercialize a gene therapeutic for patients with chronic lung disease, utilizing 4DMT’s novel discovery platform to generate optimized AAV vectors.
4DMT and ROCHE Expand Ophthalmology Partnership to Develop and Commercialize Multiple AAV Gene Therapy Products
4D Molecular Therapeutics today announced the expansion of its 2016 research agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. into a broad long-term partnership to develop and commercialize multiple ophthalmology products.
4D Molecular Therapeutics Receives $3 Million Follow-On Funding From Cystic Fibrosis Foundation Therapeutics For Gene Therapy Ind Candidate For Cystic Fibrosis