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| Tx name | NPY gene therapy |
| Description | In vivo NPY gene transfer allows for high level expression and functional efficacy limited to target region in the temporal lobe |
| Company | Neurologix |
| Status | Primate model testing |
| Overview | NPY, a 36 aa, is the most abundant neuropeptide in the brain Animals deficient in NPY show an epileptic phenotype Overexpressing NPY in the hippocampus via either germline (transgenic) or via somatic cell gene transfer (NRGX, AAV-NPY) has consistent effects on suppressing seizures in both acute (kindling, drug induced) as well as chronic spontaneous epilepsy models Acts via direct agonism on NPY Y2 receptors, which are upregulated in both animal models and human TLE Y2 receptors largely presynaptic, inhibit glutamate release Y2 receptors expressed widely and have pleiotropic actions so systemically administered agonists likely to have unwanted effects. |
| PK | |
| indications | Treatment-resistant epilepsy |
| preclin data | |
| preclin tox | |
| Phase 1 | |
| Phase 2 | |
| Phase 3 | |
| Adverse effects | clinical experience with AAV gene transfer shows not a single gene therapy related adverse event after 35 surgeries |
| publications | |
| IP | 3 key patents for AAV-Mediated Delivery of DNA to Cells of the Nervous System, Helper Functions for Recombinant Vector Production |
| Potential problems | Difficult regulatory environment. Staff turnover at the FDA leading to a more conservative approach (data supporting our PD product was much less than for NPY/Epilepsy which has 16 supporting preclinical studies) IRBs also often have a knee jerk reaction that gene therapy is both unproven and unsafe Funding two programs in parallel with limited resources and staff Epilepsy doesn’t engender as much as interest as neurodegenerative disorders from potential investors |
| Timeline | Completion of preclinical (by 2H 2010); Phase I (2010-2011) |
| Financial Status | |
| Partnerships | |
| ETP Support | grant |

| Title | Posted | |
|---|---|---|
| onfi | May 25, 2012 | |
| amy71 | ||
| non epileptic seizures from university of arizona(?) | May 25, 2012 | |
| breezie68 | ||
| Nocturnal seizures | May 25, 2012 | |
| nocturnal11 | ||
| Lamictal & Seizure questions - help??? | May 25, 2012 | |
| PPlex | ||
| Severe Epilepsy And Pregnancy | May 25, 2012 | |
| AmyBoop88 | ||
| 7 yr old seizures and autism/hearing loss/adhd, etc | May 25, 2012 | |
| jackjack | ||
| epilepsy and hormonal effects | May 25, 2012 | |
| cindyloowho | ||
| Tried Potiga yet? | May 24, 2012 | |
| tcameron | ||
| Can exercise induce seizures? | May 24, 2012 | |
| MichaelK | ||
| Lamictal Nausea, Dizzy Spells | May 24, 2012 | |
| karyliin6 | ||
| View all Forums | ||
| Title | Page Views | |
|---|---|---|
| my.epilepsy.com Updates | 20,619 | |
| epi_help | ||
| topamax and weight loss | 19,330 | |
| alexia mom | ||
| kepra | 19,244 | |
| brian mattingly | ||
| Possible cure for absence seizures | 16,388 | |
| pdl1 | ||
| How exactly do aura's feel | 14,077 | |
| WendyBendy | ||
| Sexual Side Effects | 13,524 | |
| George R | ||
| MEDICAL ALERT I.D.'s | 12,453 | |
| picnupthepcs | ||
| Over 40 Different Types Of Seizures - Revised | 10,443 | |
| spiz | ||
| electrical shock in head? | 10,083 | |
| Maggie | ||
| Weight Gain and Depakote | 9,011 | |
| galinda | ||
| View all Forums | ||
| Title | Posted | |
|---|---|---|
| Update 2012 -- lots has happened! | May 25, 2012 | |
| ninabva68 | ||
| My Blog Disappeared! Can't my seizures too? | May 24, 2012 | |
| MichaelK | ||
| SEIZURES ARE CHANGING. WHY? WHAT KIND AM I HAVING NOW? BRAIN EXPLOSION I FELT IS A MYSTERY TO ME | May 24, 2012 | |
| ROCKNROLL | ||
| Hyperventilation & epilepsy, a way to help yourself. | May 23, 2012 | |
| buteykomike | ||
| Medicine adjustments | May 23, 2012 | |
| fashionlove45 | ||
| NOT YELLING, VISUALLY IMPAIRED. POSTICTAL SHOPPING. ANYONE ELSE WITH THIS PROBLEM? TBI AND SHOPPING | May 22, 2012 | |
| ROCKNROLL | ||
| Seizures and medical marijuana | May 21, 2012 | |
| seizureprone134 | ||
| being unigue and being hypocrite | May 20, 2012 | |
| futuer poet | ||
| Coming out? | May 19, 2012 | |
| fashionlove45 | ||
| RWAY40 | May 19, 2012 | |
| rway40 | ||
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| Title | Page Views | |
|---|---|---|
| my partial complex seizures | 4,540 | |
| Zanna1211 | ||
| Topomax... The Dreaded......... | 3,787 | |
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| JudiS | ||
| How can you tell if a sleep seizure happens? | 3,545 | |
| epl_controller | ||
| Feeling Sick | 3,308 | |
| JBJ1984 | ||
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| pksmom | ||
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| Butterflygrl | ||
| TYLENOL, AEDs & SEIZURES | 2,836 | |
| cmscribbles | ||
| Nonepileptic "Events" vs. "Seizures" | 2,711 | |
| teft | ||
| newborn seizure | 2,256 | |
| Sunny_80 | ||
| View all Blogs | ||
| Title | Posted | |
|---|---|---|
| Angela-48 | May 11, 2012 | |
| ANGELA-48 | ||
| Babies with seizures | May 5, 2012 | |
| gabrielsmommy121911 | ||
| Parent and Family Support Group Newton Kansas | Mar 13, 2012 | |
| Brown67114 | ||
| No more driving | Mar 9, 2012 | |
| Amy829 | ||
| Audubon, Pennsylvania (King of Prussia area) Parents of Kids with Epilepsy Looking to connect with others in thier area | Mar 7, 2012 | |
| ajssoscia | ||
| People who have epilepsy and are parents of young children, step children or pregnant. | Feb 27, 2012 | |
| kjsdestiny | ||
| Had the worse seizure of my life last night! Complex Partial | Feb 8, 2012 | |
| jlamont | ||
| Self-Management of Medication | Feb 7, 2012 | |
| columbia.epilepsy.study | ||
| Dravet Spectrum Disorder | Feb 4, 2012 | |
| mytboy | ||
| Diving with eplilepsy | Feb 1, 2012 | |
| p59 | ||
| View all Groups | ||
| Title | Posted | |
|---|---|---|
| Imagining the past | May 25, 2012 | |
| jrgeorge | ||
| Feeling Epilepsy as a part of Life....... | May 25, 2012 | |
| Avnish | ||
| My life with epilepsy | May 24, 2012 | |
| BigTom_85 | ||
| I thought there was nothing wrong | May 24, 2012 | |
| Mendoza87 | ||
| New to this | May 23, 2012 | |
| kitkat2503 | ||
| Is it Emotional | May 23, 2012 | |
| frustrated1965 | ||
| Adjusting with the thought of epilepsy after misdiagnosis for over 20 years | May 22, 2012 | |
| goofyheather | ||
| Continuing with life | May 21, 2012 | |
| nastaran | ||
| Working my life around having epilepsy | May 21, 2012 | |
| 1505loupylou | ||
| The frustration of not knowing exaclty what is wrong... | May 20, 2012 | |
| cag122 | ||
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| Title | Page Views | |
|---|---|---|
| Jessica Roiz | 2,433 | |
| kroiz | ||
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| my brain has died a thousand deaths........... | 476 | |
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| Kelly's Life With Epilepsy | 472 | |
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| karalyeva | ||
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| dizzygal | ||
| My Daily Philosophy..... | 366 | |
| KatieFerguson88 | ||
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