This study is to determine the long-term levels of CNTF, and the clinical safety and efficacy of the NT-501 implants with the Medica Hollow Fiber Membrane (MHFM) in participants with macular telangiectasia type 2.

开始日期2025-04-01

申办/合作机构

Neurotech Pharmaceuticals, Inc.

NCT04729972 / Completed临床2期

Phase II, Multicenter, Open-label Safety Study of Bilateral NT-501 in Participants With Macular Telangiectasia Type 2

This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.

开始日期2021-08-13

申办/合作机构

Neurotech Pharmaceuticals, Inc.

[+1]

NCT04577300 / Recruiting临床2期IIT

A Randomized, Sham Controlled, Masked Phase II Study to Evaluate the Safety and Efficacy of Dual Intravitreal Implantation of NT-501 Encapsulated Cell Therapy for the Treatment of Glaucoma

To determine the safety and efficacy over 24 months of dual NT-501 CNTF encapsulated cell therapy (ECT) on visual impairment related to glaucoma.

开始日期2021-05-15

申办/合作机构

Stanford University

100 项与 Revakinagene taroretcel 相关的临床结果

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项与 Revakinagene taroretcel 相关的文献（医药）

2024-03-01·Current opinion in ophthalmology

Future directions of glaucoma treatment: emerging gene, neuroprotection, nanomedicine, stem cell, and vascular therapies

Review

作者: Ciociola, Elizabeth C. ; Fernandez, Elise ; Kaufmann, Mary ; Klifto, Meredith R.

Purpose of review:

The aim of this article is to summarize current research on novel gene, stem cell, neuroprotective, nanomedicine, and vascular therapies for glaucoma.

Recent findings:

Gene therapy using viral vectors and siRNA have been shown to reduce intraocular pressure by altering outflow and production of aqueous humor, to reduce postsurgical fibrosis with few adverse effects, and to increase retinal ganglion cell (RGC) survival in animal studies. Stem cells may treat glaucoma by replacing or stimulating proliferation of trabecular meshwork cells, thus restoring outflow facility. Stem cells can also serve a neuroprotective effect by differentiating into RGCs or preventing RGC loss via secretion of growth factors. Other developing neuroprotective glaucoma treatments which can prevent RGC death include nicotinamide, the NT-501 implant which secretes ciliary neurotrophic factor, and a Fas-L inhibitor which are now being tested in clinical trials. Recent studies on vascular therapy for glaucoma have focused on the ability of Rho Kinase inhibitors and dronabinol to increase ocular blood flow.

Summary:

Many novel stem cell, gene, neuroprotective, nanomedicine, and vascular therapies have shown promise in preclinical studies, but further clinical trials are needed to demonstrate safety and efficacy in human glaucomatous eyes. Although likely many years off, future glaucoma therapy may take a multifaceted approach.

2023-09-01·Ophthalmology science

Phase I NT-501 Ciliary Neurotrophic Factor Implant Trial for Primary Open-Angle Glaucoma

Article

作者: Satterfield, Kellie R ; Lam, Byron L ; Albini, Thomas A ; Nuñez, Mariana ; Beykin, Gala ; Goldberg, Jeffrey L

Purpose:

To assess the safety and efficacy of a ciliary neurotrophic factor (CNTF) intraocular implant on neuroprotection and neuroenhancement in glaucoma.

Design:

Open-label, prospective, phase I clinical trial.

Participants:

A total of 11 participants were diagnosed with primary open-angle glaucoma (POAG). One eye of each patient was assigned as the study (implant) eye.

Methods:

The study eye was implanted with a high-dose CNTF-secreting NT-501 implant, whereas the other eye served as a control. All patients were followed up for 18 months. Analysis was limited to descriptive statistics.

Main Outcome Measures:

Primary outcome was safety through 18 months after implantation assessed by serial eye examinations, structural and functional testing, and adverse events (AEs) recording. Parameters measured included visual acuity (VA), Humphrey visual field (HVF), pattern electroretinogram, scanning laser polarimetry with variable corneal compensation (GDx VCC), and OCT. These parameters were also used for secondary analysis of efficacy outcome.

Results:

All NT-501 implants were well tolerated with no serious AEs associated with the implant. The majority of AEs were related to the implant placement procedure and were resolved by 12 weeks after surgery. Foreign-body sensation was the most commonly reported AE and was self-limited to the postoperative period. The most common implant-related AE was pupil miosis; no patients underwent explant. Visual acuity and contrast sensitivity decreased more in fellow eyes than in study eyes (VA, -5.82 vs. -0.82 letters; and contrast sensitivity, -1.82 vs. -0.37 letters, for fellow vs. study eyes, respectively). The median HVF visual field index and mean deviation measurements worsened (decreased) in fellow eyes (-13.0%, -3.9 dB) and improved (increased) in study eyes (2.7%, 1.2 dB). Implanted eyes showed an increase in retinal nerve fiber layer thickness measured by OCT and by GDx VCC (OCT, 2.66 μm vs. 10.16 μm; and GDx VCC, 1.58 μm vs. 8.36 μm in fellow vs. study eyes, respectively).

Conclusions:

The NT-501 CNTF implant was safe and well tolerated in eyes with POAG. Eyes with the implant demonstrated both structural and functional improvements suggesting biological activity, supporting the premise for a randomized phase II clinical trial of single and dual NT-501 CNTF implants in patients with POAG, which is now underway.

Financial Disclosures:

Proprietary or commercial disclosure may be found after the references.

2021-03-01·Veterinary ophthalmology4区 · 农林科学

Medical anti‐glaucoma therapy: Beyond the drop

4区 · 农林科学

Review

作者: Miller, Paul E. ; Eaton, Joshua Seth

Abstract:

Barriers to effective medical therapy are numerous and include difficulties with effective and sustained control of intraocular pressure (IOP) and adherence to prescribed anti‐glaucoma drop regimens. In an effort to circumvent these challenges, a number of new anti‐glaucoma therapies with sustained effects have emerged. Methods for sustained delivery of prostaglandin analogs are being intensely investigated and many are in human clinical trials. Intracameral devices include the following: Allergan's Durysta™ Bimatoprost SR, Envisia Therapeutics’ ENV515 travoprost implant, Glaukos’ iDose™, Ocular Therapeutix's OTX‐TIC travoprost implant, and Santen's polycaprolactone implant with PGE2‐derivative DE‐117. Other prostaglandin‐based technologies include Allergan's bimatoprost ring (placed in the conjunctival fornix), Ocular Therapeutics’ OTX‐TP intracanalicular travoprost implant, subconjunctival latanoprost in a liposomal formulation, and the PGE2 derivative PGN 9856‐isopropyl ester that is applied to the periorbital skin. Exciting breakthroughs in gene therapy include using viral vectors to correct defective genes such as MYOC or to modulate gonioimplant fibrosis, CRISPR technology to edit MYOC or to alter aquaporin to reduce aqueous humor production, and siRNA technology to silence specific genes. Stem cell technology can repopulate depleted tissues or, in the case of Neurotech's Renexus® NT‐501 intravitreal implant, serve as a living drug delivery device that continuously secretes neurotrophic factors. Other unique approaches involve nanotechnology, nasal sprays that deliver drug directly to the optic nerve and noninvasive alternating current stimulation of surviving cells in the optic nerve. Over time these modalities are likely to challenge the preeminent role that drops currently play in the medical treatment of glaucoma in animals.

项与 Revakinagene taroretcel 相关的新闻（医药）

2024-06-20

·BioSpace

Neurotech Pharmaceuticals, Inc. Receives Priority Review of Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) as a Treatment for Macular Telangiectasia Type 2 (MacTel)

- Priority review granted with a PDUFA goal date set for December 17, 2024. CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., an innovator in sustained drug delivery for chronic retinal diseases, announces that the U.S. Food and Drug Administration (FDA), has determined that the Biologic License Application (BLA) for NT-501, an investigational encapsulated cell therapy for the treatment of MacTel, is sufficiently complete to permit a substantive review. The application has a prescription drug user fee act (PDUFA) goal date of December 17, 2024. MacTel is a progressive, neurodegenerative disease of the retina that results in the deterioration of central vision, significantly impacting patients' quality of life. NT-501 is an ocular implant designed to deliver sustained therapeutic doses of ciliary neurotrophic factor (CNTF) directly to the retina to slow the progression of the disease. “This is a significant achievement for Neurotech,” said Richard Small, Chief Executive Officer. “I would like to express my gratitude to our employees for reaching this important milestone.” About Macular Telangiectasia (MacTel) MacTel, or idiopathic juxtafoveal macular telangiectasia, is a rare neurodegenerative disease with characteristic localized retinal degeneration with secondary alterations of the retinal vasculature.1 MacTel typically affects both eyes and causes deterioration in central vision impacting patients’ quality of life. About Encapsulated Cell Therapy (ECT) The ECT platform is a cell-based delivery system that was designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic retinal diseases. This versatile platform consists of a small, semi-permeable capsule containing proprietary allogeneic RPE cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment. The capsule is surgically inserted into the patient’s vitreous and sutured to the sclera during an outpatient procedure. Once in place, the capsule's semi-permeable membrane allows essential nutrients to enter, while also permitting the therapeutic proteins to exit into the vitreous, providing targeted and continuous treatment. Simultaneously, the membrane protects the encapsulated RPE cells from the host's immune system, ensuring their long-term survival and functionality. About NT-501 Implant NT-501 leverages our ECT platform to deliver CNTF for the treatment of chronic retinal diseases, such as MacTel. CNTF is a potent neuroprotective protein that promotes the survival and maintenance of photoreceptors. This targeted therapy provides sustained delivery of CNTF, aiming to slow retinal degeneration and potentially improve long-term visual outcomes for patients. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private, clinical stage biotechnology company and an innovator in sustained drug delivery for chronic retinal diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the back of the eye. For more information about us, please visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2.

细胞疗法优先审批

2023-09-13

·BioSpace

Alex Gorsky Joins Neurotech’s Board as Lead Director

CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., a private clinical stage biotech company, announced today that Alex Gorsky has been elected to Neurotech’s board as Lead Director. Gorsky is the former Chairman and CEO of Johnson & Johnson. This press release features multimedia. View the full release here: Alex Gorsky (Photo: Business Wire) Under Gorsky’s leadership, Johnson & Johnson grew to become the world’s largest diversified healthcare company and one of the foremost innovators in research and development for emerging health technologies. His influence has shaped both the healthcare landscape and the greater business community through his work as a member of the Business Roundtable, the Business Council, and many industry groups. He currently sits on the boards of Apple, IBM, JPMorgan Chase, and the Travis Manion Foundation, as well as the Wharton School of the University of Pennsylvania Board of Advisors. “I am delighted to welcome Alex to Neurotech’s Board of Directors,” said Jim Mazzo, Neurotech’s Executive Chairman. “As a visionary in patient focused healthcare, his expertise and enthusiasm for innovative technology will greatly enrich Neurotech’s mission to revolutionize treatments for various ocular diseases using our encapsulated cell therapy.” Mr. Gorsky said, “I am honored to join Neurotech’s board and look forward to using my experience to help the company navigate in a rapidly evolving environment. I am inspired by the Lowy family’s long-term commitment. This unique scientific approach and novel technology for the treatment of chronic eye diseases has the potential to provide access to important health options for patients around the world.” About Macular telangiectasia type 2 Macular telangiectasia type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 MacTel typically affects both eyes and causes a gradual deterioration in central vision. About NT-501 Implant Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel. About Encapsulated Cell Therapy Encapsulated Cell Therapy (ECT) is an investigational first-in-class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that is genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins. The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance, while reducing the treatment burden with one surgical procedure that can deliver the drug for at least 2 years. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, Encapsulated Cell Therapy (ECT), enables continuous production of therapeutic proteins to the eye. Neurotech is currently studying in the clinic ECT candidates to treat Macular telangiectasia type 2 and glaucoma. To learn more, visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2. Prog. Retin. Eye Res. 34: 49-77.

细胞疗法高管变更临床3期

2023-01-10

·BioSpace

Neurotech Pharmaceuticals, Inc. Expands Executive Team with the Appointment of Scott Hunter as Chief Commercial Officer

CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., a clinical stage biotech company, today announced the expansion of its executive team with the appointment of Scott Hunter as Chief Commercial Officer bringing two decades of eyecare experience across both rare disease and specialty markets. This press release features multimedia. View the full release here: Scott Hunter Joins Neurotech Pharmaceuticals as Chief Commercial Officer (Photo: Business Wire) “Scott’s appointment as the Company’s first Chief Commercial Officer marks an important milestone for Neurotech. Scott will be instrumental in helping us develop the commercial strategy and establish our commercial infrastructure,” said Richard Small, Chief Executive Officer of Neurotech. “His proven success of bringing products to market that address unmet needs is a perfect fit with our vision and culture, which is dedicated to slowing disease progression while helping patients maintain vision and quality of life.” “I am extremely excited to join the Neurotech team and build the commercial organization” stated Scott Hunter. “Starting with science behind NT-501, I believe Neurotech can drastically change the management of sight-threatening retinal conditions such as MacTel.” Scott joins Neurotech with a demonstrated track record in global rare and specialty eyecare experience. He most recently worked at Novartis where he was the Global Marketing Head for Luxturna (voretigene neparvovec-rzyl), responsible for advancing the global strategy for the first commercially approved ocular gene therapy. Before Novartis, Scott was at Shire, where he was instrumental in the build-out of the U.S. ophthalmics franchise and launch of Xiidra, culminating in the divestiture to Novartis for up to $5.3 billion. Prior he spent 12 years at Bausch & Lomb, where he held roles of increasing responsibility across research and development (R&D) and marketing. Scott earned his M.S. in Pharmacoeconomics from the University of Florida and his M.B.A. from the University of Tampa after completing his B.S. in Biology at the University of South Florida. About Macular telangiectasia type 2 Macular telangiectasia type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 MacTel typically affects both eyes and causes a gradual deterioration in central vision. About NT-501 Implant Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel. About Encapsulated Cell Therapy Encapsulated Cell Therapy (ECT) is an investigational first-in-class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that has been genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins. The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance and reducing treatment burden with one surgical procedure that can deliver drug for at least 2 years. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the eye. Neurotech is currently studying in the clinic ECT candidates to treat Macular telangiectasia type 2 and glaucoma. To learn more, visit . Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2. Prog. Retin. Eye Res. 34: 49-77.

临床3期细胞疗法高管变更基因疗法

100 项与 Revakinagene taroretcel 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
2型黄斑毛细血管扩张症	申请上市	美国	Neurotech Pharmaceuticals, Inc.	2024-06-20
视网膜毛细血管扩张	临床3期	澳大利亚	Neurotech USA, Inc.	2018-01-19
视网膜色素变性	临床3期	美国	Neurotech USA, Inc.	2007-04-16
色觉障碍	临床2期	美国	Neurotech USA, Inc.	2012-07-07
全色盲1型	临床2期	美国	National Eye Institute	2012-07-01
Usher综合征	临床2期	美国	Neurotech USA, Inc.	2012-01-20
地图样萎缩	临床2期	美国	Neurotech USA, Inc.	-
青光眼	临床1期	美国	Neurotech USA, Inc.	-
青光眼	临床1期	美国	University of Miami	-
视神经疾病	临床1期	美国	Neurotech USA, Inc.	-

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ARVO2023	N/A	-	-	NT-501 Implant	膚廠衊構醖憲夢窪淵顧(範廠壓壓築鏇鏇餘衊憲) = 廠艱鏇壓艱鹽廠顧淵築願醖蓋積窪選鹹觸膚遞 (齋獵簾襯襯選膚憲簾簾 )	-	2023-04-23
Biospace 人工标引	临床3期	2型黄斑毛细血管扩张症	113	NT-501	糧顧鹽壓範鹹製鏇遞蓋(憲廠願網簾選衊艱淵構) = 壓鹽淵憲襯鹽衊鬱繭鬱夢築繭鬱鹹鏇齋膚製繭 (壓壓獵衊選鹽糧鏇鬱鏇 ) 更多	积极	2022-11-02
				Sham Procedure	-
NCT03316300 (Protocol A, Biospace) 人工标引	临床3期	2型黄斑毛细血管扩张症	115	NT-501	鹽網鏇艱淵鏇遞糧構夢(鹽膚糧淵憲糧膚鏇簾選) = 繭選網膚積鹽繭齋膚齋積築艱鑰鹽積窪齋築糧 (鬱衊憲築鹹鏇簾鹹蓋獵 )	积极	2022-11-02
				Sham Procedure	-
NCT03316300 (Protocol A, Pubmed) 人工标引	临床3期	2型黄斑毛细血管扩张症 \| 黄斑变性	-	NT-501	餘鹹膚衊鏇憲願簾製鏇(遞艱築築醖餘築廠繭繭) = 壓壓淵衊蓋選觸憲網膚衊獵遞醖製艱憲鏇餘觸 (繭顧選構夢簾鬱積鏇廠, 0.19)	积极	2021-03-15
				untreated	餘鹹膚衊鏇憲願簾製鏇(遞艱築築醖餘築廠繭繭) = 鏇憲選壓鹹窪製獵範遞衊獵遞醖製艱憲鏇餘觸 (繭顧選構夢簾鬱積鏇廠, 0.11)