4DMT operates as a clinical-stage genetic medicines company focused on inventing and developing genetic medicines to treat large market diseases in ophthalmology, pulmonology and cardiology. It develops genetic medicines using its proprietary invention platform, Therapeutic Vector Evolution. It combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences. Its product design, development and manufacturing engine create a valuable and diverse product pipeline. The company is currently advancing five clinical-stage and two preclinical product candidates, each tailored to address rare and large market diseases. 4DMT was founded by David H. Kirn, Melissa Kotterman, Theresa Janke, and David Schaffer on September 12, 2013 and is headquartered in Emeryville, CA.
相关临床试验
10
5 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2013
进行中（未招募）
5
50.0%
招募中
4
40.0%
终止
1
10.0%
暂无批准数据
- At ASRS 2026 in Montreal, durable treatment strategies for wet AMD dominated research, with gene therapy, intravitreal implant and optogenetic data presented across four days. - Ocular Therapeutix reported phase 3 SOL-1 results showing a single AXPAXLI injection maintained BCVA in 74.1% of patients at Week 36 versus 55.8% with monthly aflibercept. - Nanoscope Therapeutics presented 3-year REMAIN data showing durable BCVA gains of about 3 ETDRS lines with MCO-010 in retinitis pigmentosa, alongside a rolling BLA submission. - Belite Bio's phase 3 DRAGON trial of oral tinlarebant met its primary endpoint with a 36% reduction in retinal lesion growth versus placebo in Stargardt disease.
- 4D Molecular Therapeutics announced positive interim Phase 1 data for 4D-710, showing clinically meaningful lung function improvements measured by ppFEV1 and LCI2.5 with follow-up through one year. - The gene therapy demonstrated durable CFTR transgene expression within target therapeutic range and was well-tolerated, with the 2.5E14 vg dose selected for Phase 2 development. - 4D-710 represents the first genetic medicine to successfully deliver and express the CFTR transgene throughout airways of cystic fibrosis patients after aerosol delivery. - The therapy is designed as a variant-agnostic treatment that could become foundational therapy for many CF patients regardless of their specific CFTR variant.
- Alkeus Pharmaceuticals has appointed renowned retina specialist Carlos Quezada-Ruiz, M.D., F.A.S.R.S., as Chief Medical Officer to lead clinical development of gildeuretinol for Stargardt disease. - The company is accelerating preparations for a global Phase 3 registrational study of gildeuretinol, an oral therapy with breakthrough designation targeting ABCA4-related retinal degeneration. - Dr. Quezada-Ruiz brings extensive experience from 4D Molecular Therapeutics and Genentech/Roche, where he led development of treatments for inherited and neovascular retinal diseases. - Seemi Khan, M.D., the previous Chief Medical Officer, has been promoted to the newly created role of Chief Development and Strategy Officer.
- Tyrosine kinase inhibitors EYP-1901 and OTX-TKI have demonstrated preliminary results similar to aflibercept 2 mg in treating wet age-related macular degeneration. - Multiple gene therapy programs including ABBV-RGX-314, 4D-150, and ADVM-022 are currently under investigation for neovascular AMD treatment. - Vorolanib is designed for 6-month dosing intervals, potentially reducing treatment burden for wet AMD patients. - Experts anticipate numerous clinical readouts in the next year or two that could expand treatment options for neovascular AMD patients.
- 4D Molecular Therapeutics has appointed Kristian Humer as Chief Financial Officer to lead financial strategy as the company advances multiple Phase III programs and prepares for commercial readiness. - Humer brings over two decades of experience in corporate finance and healthcare investment banking, having previously served as Managing Director at Citi's Healthcare Investment Banking Group and CFO at publicly traded companies Foghorn Therapeutics and Viridian Therapeutics. - The appointment comes as 4DMT's lead product candidate 4D-150 progresses through Phase III development for wet age-related macular degeneration, with a second indication in diabetic macular edema. - 4D-150 is designed as a backbone therapy providing multi-year sustained delivery of anti-VEGF agents with a single intravitreal injection, potentially reducing treatment burden for blinding retinal vascular diseases.
- ResearchAndMarkets.com has released a comprehensive report analyzing licensing deals in biotechnology from 2020 to 2025, providing insights into deal trends and structures. - The report covers hundreds of companies including major players like AbbVie, ACADIA Pharmaceuticals, and emerging biotech firms across various therapeutic areas and technology types. - Key features include financial terms analysis covering upfront payments, milestones, and royalties, plus access to actual contract documents for due diligence purposes. - The analysis enables benchmarking of transaction values and identification of the most active dealmakers in the biotechnology licensing landscape.
- 4D Molecular Therapeutics announced a strategic partnership with Otsuka Pharmaceutical for exclusive development and commercialization rights of 4D-150 in the Asia-Pacific region, including Japan, China, and Australia. - The agreement includes an $85 million upfront payment and at least $50 million in cost sharing over three years, with potential milestone payments reaching up to $336 million plus tiered double-digit royalties. - 4D-150 is designed as a potentially transformative gene therapy providing multi-year sustained anti-VEGF delivery with a single intravitreal injection for wet AMD and diabetic macular edema. - The partnership aims to address significant unmet medical needs in the APAC region, where wet AMD affects over 4 million patients in major markets and DME represents a large underserved population.
- The Cystic Fibrosis Foundation will provide up to $11 million in additional funding to 4D Molecular Therapeutics to accelerate development of 4D-710, a gene therapy for cystic fibrosis lung disease. - Phase 2 enrollment in the AEROW clinical trial is currently underway with 2.5E14 vg selected as the anticipated pivotal and commercial dose for the variant-agnostic genetic medicine. - The funding supports redosing studies and Phase 3 readiness, with interim Phase 1 data including functional durability results expected by year-end 2025. - 4D-710 represents the first known genetic medicine to demonstrate successful CFTR transgene delivery and expression throughout CF patient airways via aerosol delivery.
- Phase 1/2 SPECTRA trial of 4D-150 demonstrates consistent efficacy and safety profile in treating diabetic macular edema, with sustained improvements in visual acuity. - The gene therapy treatment significantly reduced the need for supplemental injections, potentially offering a more convenient treatment option for DME patients. - H.C. Wainwright analyst maintains 'Buy' rating with $36 price target, citing strong potential in both ophthalmology and pulmonology programs.
• REGENXBIO's ABBV-RGX-314 gene therapy significantly reduced the need for anti-VEGF injections in patients with bilateral wet AMD. • A Phase 2 substudy demonstrated a 97% decrease in annualized anti-VEGF treatment burden after ABBV-RGX-314 administration in the fellow eye. • 78% of patients were completely free of anti-VEGF injections at 9 months post-treatment, with sustained visual acuity and retinal thickness. • ABBV-RGX-314 was well-tolerated, marking a potential milestone for gene therapy in treating common retinal diseases like wet AMD.
