Neurotech Pharmaceuticals, Inc., a Lincoln, Rhode Island based firm, has announced that the FDA has granted Fast Track designations for the firm’s lead product candidate, the NT-501, for treatment of visual loss in two indications: retinitis pigmentosa (RP) and dry form of age-related macular degeneration (dry AMD). Such designations should allow the company to accelerate clinical development of its continuous, long-term release formulation of the therapeutic protein Ciliary Neurotrophic Factor (CNTF), designed to be released into the vitreous body from a proprietary Encapsulated Cell Technology (ECT) device.
More about Neurotech’s technology platform and the NT-501:
Neurotech’s core technology platform is Encapsulated Cell Technology (ECT), a unique technology that allows for the long term, sustained delivery of therapeutic factors to the back of the eye.
ECT implants consist of cells that have been genetically modified to produce a desired therapeutic factor that are encapsulated in a section of semi-permeable hollow fiber membrane. The diffusive characteristics of the hollow fiber membrane are designed to promote long-term cell survival by allowing influx of oxygen and nutrients while simultaneously preventing direct contact of the encapsulated cells with the cellular and molecular elements of the immune system. The cells continuously produce the therapeutic protein which diffuses out of the implant at the target site. ECT therefore enables the controlled, continuous delivery of therapeutic factors directly to the retina, bypassing the blood-retina barrier. Long-term protein delivery (18 months) in the vitreous cavity of the eye has consistently been achieved when ECT devices containing human cells genetically engineered to secrete CNTF have been implanted in a highly disparate mammalian species (rabbits). In addition, the implants can be retrieved, providing an added level of safety as well as the ability to reverse or adjust therapy, if needed.
ECT based products can be tailored to address the three main clinical manifestations of retinal diseases: degeneration of photoreceptors and/or ganglion cells in the neural retina, vascular proliferation and inflammation. A number of proteins have been discovered in the field of ophthalmology that possess powerful neurotrophic, anti-angiogenic and anti-inflammatory properties. These proteins have the potential to significantly slow or halt disease processes in the eye. ECT represents a unique platform for the safe and effective delivery of various factors for the treatment of these chronic ophthalmic diseases as follows:neurotrophic factors for the treatment of retinal degeneration in Retinitis Pigmentosa (RP), Geographic Atrophy (serious condition associated with the Dry form of Age-related Macular Degeneration), Glaucoma, Retinal Vein Occlusion and others. anti-angiogenic factors for the treatment of vascular proliferation in Diabetic Retinopathy and the Wet form of AMD, and for the treatment of abnormal vascular permeability for various forms of Macular Edema. anti-inflammatory factors for the treatment of Ocular Inflammations (Uveitis) The current product is 6 mm in length and consists of genetically-modified human cells packaged in a semi-permeable hollow fiber membrane with a suture loop at one end to anchor the implant to the sclera. In contrast to gene therapy, ECT does not modify the host genome. The implant is surgically placed in the vitreous body. The implant is sutured in a manner that allows for its retrieval when desired. The surgical procedure is performed as an out-patient procedure in about 20 minutes…
… NT-501, consists of encapsulated human cells genetically modified to secrete ciliary neurotrophic factor (CNTF). NT-501 is designed to continually deliver a low, safe and therapeutic dose of CNTF, a well-established neurotrophic factor, into the back of the eye. The Company believes that CNTF activates dying retinal photoreceptors and protects them from degeneration.
Product page: NT-501…
ECT Technology…
Press release: Neurotech Granted Fast Track Designations from the FDA for NT-501 in Two Indications: Retinitis Pigmentosa and Dry Age-Related Macular Degeneration…
(hat tip: Technology Review)