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NEUROGENE INC

16.97

+1.5710.19%

Post-market: 16.970.00000.00%16:05 EDT

Volume:199.78K

Turnover:3.37M

Market Cap:242.03M

PE:-3.96

High:17.45

Open:15.54

Low:15.54

Close:15.40

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Overview Company News Filings

Neurogene Q3 Net Income USD -20.217 Million

THOMSON REUTERS

Press Release: Neurogene Reports Third Quarter 2024 Financial Results and Highlights Recent Updates

Neurogene Shares Down 35.1% After Co to Stop Giving High Dose of Gene Therapy to Patients in Study as It Investigates Possible Side Effect

THOMSON REUTERS

Neurogene Shares Down 20.5% Premarket After Co to Stop Giving High Dose of Gene Therapy to Patients in Study as It Investigates Possible Side Effect

THOMSON REUTERS

BUZZ-Neurogene drops after pausing part of gene therapy study due to serious side effect

Neurogene Receives FDA Approval to Proceed with Low-Dose Trial for Rett Syndrome Therapy

MT Newswires Live

Neurogene Drops High-Dose Cohort of Gene-Therapy Study for Rett Syndrome

Neurogene provides update on NGN-401 clinical trial for Rett syndrome

Neurogene Inc - No Other Treatment-Related Saes Reported in Ngn-401 Trial

THOMSON REUTERS

Neurogene Inc - Pauses High-Dose Cohort Enrollment in Ngn-401 Trial

THOMSON REUTERS

Neurogene Inc - Participant in Critical Condition After Ngn-401 Dose

THOMSON REUTERS

Neurogene Provides Update on Ngn-401 Gene Therapy Clinical Trial for Rett Syndrome

THOMSON REUTERS

Neurogene Inc - Reports Serious Adverse Event in Ngn-401 Trial Participant

THOMSON REUTERS

Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

Dow Dips Over 200 Points; Home Depot Posts Upbeat Earnings

Neurogene (NGNE) Receives a Buy from Stifel Nicolaus

Investor Sentiment Muted as Inflation Report Looms, Driving Quiet Premarket Action for US Equity Futures

MT Newswires Live

Top Premarket Decliners

MT Newswires Live

Neurogene Is Maintained at Outperform by Baird

Neurogene Shares Drop 35% on Rett Syndrome Gene Therapy Trial Data

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Expects to provide an update on registrational trial design in first half of 2025. Strong cash position provides runway into the second half of 2027. Neurogene Inc. , a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced third quarter 2024 financial results and highlighted recent corporate updates.Third Quarter 2024 and Recent Highlights, and Anticipated Milestones. Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome. NGN-401 is currently being evaluated in an ongoing, open-label Phase 1/2 trial. Key updates include:. Announced today an update to the NGN-401 clinical trial. Following a treatment-related serious adverse event  in the. third participant dosed with 3E15 vg, Neurogene paused further use. of the 3E15 vg dose and does not plan to enroll any further. 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effect","url":"https://stock-news.laohu8.com/highlight/detail?id=2484210076","media":"Reuters","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484210076?lang=en_us&edition=fundamental","pubTime":"2024-11-18 20:46","pubTimestamp":1731933990,"startTime":"0","endTime":"0","summary":"** Drug developer Neurogene's    shares fall 19% to $28 premarket   ** Company says it has paused testing high-dosage version of its experimental gene therapy after reporting a serious treatment related side-effect in one of the participants   ** Neurogene says the participant is in critical condition due to hyperinflammatory syndrome, a rare and life-threatening immune response   ** The gene therapy, NGN-401, is being developed to treat Rett syndrome, a rare genetic neurological disorder that occurs most often in girls and affects their ability to speak, walk, eat, and even breathe easily   ** Company says the U.S. Food and Drug Administration has allowed the early-mid-stage study to continue testing patients with the low-dosage version   ** As of last close, stock up 78% YTD ","market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.rkd.refinitiv.com/api/News/News.svc/REST/News_1/RetrieveStoryML_1","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"-1","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":null,"symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484621495","title":"Neurogene Receives FDA Approval to Proceed with Low-Dose Trial for Rett Syndrome Therapy","url":"https://stock-news.laohu8.com/highlight/detail?id=2484621495","media":"MT Newswires Live","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484621495?lang=en_us&edition=fundamental","pubTime":"2024-11-18 20:39","pubTimestamp":1731933564,"startTime":"0","endTime":"0","summary":"Neurogene  said Monday that the US Food and Drug Administration has allowed the company to continue an early-stage trial using a lower dose of NGN-401, the company's intended gene therapy for Rett syndrome, a rare genetic neurological disorder.The company said it had paused the higher-dose cohort of the study following a previously disclosed emerging treatment-related serious adverse event in a trial participant. It also said it does not plan to enroll any more participants at the higher-dose level.So far, the company said it had not received reports of other treatment-related serious adverse events in the trial. However, it said that it no longer expects to complete enrollment of participants in the low-dose cohort in Q4.Shares of the company fell more than 18% in premarket activity.Price: 28.00, Change: -6.51, Percent Change: -18.88","market":"sg","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.mtnewswires.com/","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"1","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"mtnewswires_news","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484997621","title":"Neurogene Drops High-Dose Cohort of Gene-Therapy Study for Rett Syndrome","url":"https://stock-news.laohu8.com/highlight/detail?id=2484997621","media":"Dow Jones","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484997621?lang=en_us&edition=fundamental","pubTime":"2024-11-18 20:34","pubTimestamp":1731933240,"startTime":"0","endTime":"0","summary":"Neurogene is proceeding with a scaled-back study of its proposed NGN-401 gene therapy for the rare genetic neurological disorder Rett syndrome after a serious adverse event left a study participant in grave condition.Neurogene on Monday said the U.S. Food and Drug Administration has completed a review of the safety data for NGN-401 and is allowing the New York company to proceed with the Phase 1/2 trial study using the low-dose cohort.Neurogene last week reported that it had become aware of an emerging treatment-related serious adverse event in a participant in the study's high-dose cohort, and the news sent its share price tumbling 44%.The company on Monday said the patient experienced signs of a systemic hyperinflammatory syndrome, a rare and life-threatening immune response that has been reported with systemic exposure to high doses of a benign virus used to deliver genetic material, and is in critical condition.Neurogene said it doesn't plan to enroll any further study participants","market":"sg","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"-2","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":null,"symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484626525","title":"Neurogene provides update on NGN-401 clinical trial for Rett syndrome","url":"https://stock-news.laohu8.com/highlight/detail?id=2484626525","media":"TIPRANKS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484626525?lang=en_us&edition=fundamental","pubTime":"2024-11-18 20:00","pubTimestamp":1731931239,"startTime":"0","endTime":"0","summary":"Neurogene (NGNE) announced an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett s...","market":"fut","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":{"source":"tipranks_highlight","url":"https://www.tipranks.com/news/the-fly/neurogene-provides-update-on-ngn-401-clinical-trial-for-rett-syndrome?utm_source=itigerup.com&utm_medium=referral","rn_cache_url":null,"directOrigin":true},"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.tipranks.com/news/the-fly/neurogene-provides-update-on-ngn-401-clinical-trial-for-rett-syndrome?utm_source=itigerup.com&utm_medium=referral","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"tipranks_highlight","symbols":["NGNE","BK4139"],"gpt_icon":0},{"id":"2484625587","title":"Neurogene Inc - No Other Treatment-Related Saes Reported in Ngn-401 Trial","url":"https://stock-news.laohu8.com/highlight/detail?id=2484625587","media":"THOMSON REUTERS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484625587?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":null,"market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20241118:nTUA28Z6BV:3","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"reuters_en_live","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484625582","title":"Neurogene Inc - Pauses High-Dose Cohort Enrollment in Ngn-401 Trial","url":"https://stock-news.laohu8.com/highlight/detail?id=2484625582","media":"THOMSON REUTERS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484625582?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":null,"market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20241118:nTUA28Z6BV:4","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"reuters_en_live","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484625563","title":"Neurogene Inc - Participant in Critical Condition After Ngn-401 Dose","url":"https://stock-news.laohu8.com/highlight/detail?id=2484625563","media":"THOMSON REUTERS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484625563?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":null,"market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20241118:nTUA28Z6BV:5","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"reuters_en_live","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484581625","title":"Neurogene Provides Update on Ngn-401 Gene Therapy Clinical Trial for Rett Syndrome","url":"https://stock-news.laohu8.com/highlight/detail?id=2484581625","media":"THOMSON REUTERS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484581625?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":null,"market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20241118:nTUA28Z6BV:1","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"reuters_en_live","symbols":["NGNE","BK4139"],"gpt_icon":0},{"id":"2484255806","title":"Neurogene Inc - Reports Serious Adverse Event in Ngn-401 Trial Participant","url":"https://stock-news.laohu8.com/highlight/detail?id=2484255806","media":"THOMSON REUTERS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484255806?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":null,"market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20241118:nTUA28Z6BV:2","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"reuters_en_live","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2484625503","title":"Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome","url":"https://stock-news.laohu8.com/highlight/detail?id=2484625503","media":"Business Wire","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2484625503?lang=en_us&edition=fundamental","pubTime":"2024-11-18 19:45","pubTimestamp":1731930300,"startTime":"0","endTime":"0","summary":"NEW YORK, November 18, 2024----Neurogene Inc. , a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome.In a commitment to full transparency with the U.S. Food and Drug Administration , Neurogene proactively engaged with the FDA under the START program following the Company’s notification of the SAE. The FDA completed a review of the safety data for NGN-401 and allowed Neurogene to proceed with the Phase 1/2 trial using the 1E15 vg dose . Neurogene paused further use of the 3E15 vg dose  upon initial notification of the SAE and does not plan to enroll any further participants at the 3E15 vg dose level.Neurogene no longer anticipates completing enrollment in the 1E15 vg cohort  of NGN-401 in the fourth quarter of 2024 as the Company updates the protocol to reflect","market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://finance.yahoo.com/news/neurogene-provides-ngn-401-gene-114500670.html","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"yahoofinance","symbols":["BK4139","NGNE"],"gpt_icon":0},{"id":"2483053486","title":"Dow Dips Over 200 Points; Home Depot Posts Upbeat Earnings","url":"https://stock-news.laohu8.com/highlight/detail?id=2483053486","media":"Benzinga","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2483053486?lang=en_us&edition=fundamental","pubTime":"2024-11-13 03:31","pubTimestamp":1731439884,"startTime":"0","endTime":"0","summary":" ","market":"hk","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":["earning"],"language":"en","translate_title":"","themeId":"fa9ad46d4e4a3296a8869c884a553ce7","theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.benzinga.com/24/11/41916474/dow-dips-near-200-points-home-depot-posts-upbeat-earnings","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"1","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":null,"symbols":["PGY","NGNE","GNS","PCOM","TSHA","AZI","HD"],"gpt_icon":1},{"id":"2482608822","title":"Neurogene (NGNE) Receives a Buy from Stifel Nicolaus","url":"https://stock-news.laohu8.com/highlight/detail?id=2482608822","media":"TIPRANKS","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2482608822?lang=en_us&edition=fundamental","pubTime":"2024-11-12 22:16","pubTimestamp":1731420983,"startTime":"0","endTime":"0","summary":"In a report released today, Paul Matteis from Stifel Nicolaus maintained a Buy rating on Neurogene (NGNE – Research Report), with a price target of...","market":"us","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":{"source":"tipranks_highlight","url":"https://www.tipranks.com/news/blurbs/neurogene-ngne-receives-a-buy-from-stifel-nicolaus?utm_source=itigerup.com&utm_medium=referral","rn_cache_url":null,"directOrigin":true},"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.tipranks.com/news/blurbs/neurogene-ngne-receives-a-buy-from-stifel-nicolaus?utm_source=itigerup.com&utm_medium=referral","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"1","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"tipranks_highlight","symbols":["BK4139","BK4127","NGNE","SF"],"gpt_icon":0},{"id":"2482707801","title":"Investor Sentiment Muted as Inflation Report Looms, Driving Quiet Premarket Action for US Equity Futures","url":"https://stock-news.laohu8.com/highlight/detail?id=2482707801","media":"MT Newswires Live","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2482707801?lang=en_us&edition=fundamental","pubTime":"2024-11-12 21:49","pubTimestamp":1731419363,"startTime":"0","endTime":"0","summary":"US equity futures were flat before Tuesday's opening bell, with sentiment stabilizing after last week's election-fueled rally, while traders looked ahead to the consumer inflation report.The Dow Jones Industrial Average futures gained 0.2%, S&P 500 futures declined 0.1%, and Nasdaq futures flatlined.The Consumer Price Index for October is scheduled for release on Wednesday.Oil prices were higher, with front-month global benchmark North Sea Brent crude up 0.7% at $72.34 per barrel and US West Texas Intermediate crude up 0.8% at $68.56 per barrel.In other world markets, Japan's Nikkei closed 0.4% lower, Hong Kong's Hang Seng ended 2.8% lower, and China's Shanghai Composite finished 1.4% lower. Meanwhile, UK's FTSE 100 fell 0.9% and Germany's DAX index was down 1.1% in Europe's early afternoon session.In equities, shares of Shopify  were 19% higher pre-bell after the company posted higher Q3 revenue that topped analysts' estimates. Bridger Aerospace  shares rose 14% after the company repo","market":"nz","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.mtnewswires.com/","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"mtnewswires_news","symbols":["BK4566","BK4116","NGNE","UDOW","BK4139","SDOW","PSQ","BK4548","BAER","BK4532","DXD","LU2357305700.SGD","LU1720051108.HKD","BK4585","EQR","QID","LU1720051017.SGD","DOG","BK4524","TQQQ","DDM",".SPX","DJX",".DJI","BK4588","BK4137","BK4551","BK4528","LU1548497426.USD","MNQmain","LU1861558580.USD","BK4215","LU2265009873.SGD",".IXIC","SQQQ","QLD","NQmain","LU0390134368.USD","LU1861559042.SGD","LU1804176565.USD","SHOP"],"gpt_icon":1},{"id":"2482709507","title":"Top Premarket Decliners","url":"https://stock-news.laohu8.com/highlight/detail?id=2482709507","media":"MT Newswires Live","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2482709507?lang=en_us&edition=fundamental","pubTime":"2024-11-12 20:56","pubTimestamp":1731416174,"startTime":"0","endTime":"0","summary":"Neurogene (NGNE) shares slumped 34% pre-bell Tuesday, a day after the company said it will not move forward with its Batten disease gene therapy program.1847 Holdings (EFSH) shares fell 30%, paring Monday's rally.General American Investors (GAM) shares were down 18% following a muted session.","market":"sh","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":[],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://www.mtnewswires.com/","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"0","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":"mtnewswires_news","symbols":["NGNE","BK4206","BK4139","EFSH","GAM","BK4590"],"gpt_icon":0},{"id":"2482270693","title":"Neurogene Is Maintained at Outperform by Baird","url":"https://stock-news.laohu8.com/highlight/detail?id=2482270693","media":"Dow Jones","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2482270693?lang=en_us&edition=fundamental","pubTime":"2024-11-12 20:43","pubTimestamp":1731415380,"startTime":"0","endTime":"0","summary":"Ratings actions from Benzinga: https://www.benzinga.com/quote/NGNE/analyst-ratings \n\n\n \n\n\n  (END) Dow Jones Newswires\n\n\n  November 12, 2024 07:43 ET (12:43 GMT)","market":"sg","thumbnail":null,"type":0,"news_type":0,"thumbnails":[],"rights":null,"property":["rate"],"language":"en","translate_title":"","themeId":null,"theme_name":"","theme_type":"","isJumpTheme":false,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","is_publish_highlight":false,"source_rank":0,"column":"","sentiment":"1","news_top_title":null,"news_tag":"","news_rank":0,"length":0,"strategy_id":0,"source":null,"symbols":["NGNE"],"gpt_icon":0},{"id":"2482708786","title":"Neurogene Shares Drop 35% on Rett Syndrome Gene Therapy Trial Data","url":"https://stock-news.laohu8.com/highlight/detail?id=2482708786","media":"GuruFocus.com","labels":[],"top":-1,"itemType":null,"share":"https://ttm.financial/m/news/2482708786?lang=en_us&edition=fundamental","pubTime":"2024-11-12 19:58","pubTimestamp":1731412710,"startTime":"0","endTime":"0","summary":"Neurogene  shares fell 35% in Tuesday's pre-market trading following the release of interim results from a phase 1/2 trial for NGN-401, its gene therapy candidate targeting Rett syndrome.The study included pediatric patients given low and high dosages of NGN-401. All four of the low-dose group members exhibited improvement, scoring 2 on the Clinical Global Impression Scale of Improvement, thereby indicating significant change from baseline. The business also reports that these individuals showed a 28% to 52% improvement on the Rett Syndrome Behavior Questionnaire.Nonetheless, consistent with recognized dangers of AAV  gene treatments, one of the two patients in the high-dose group had a major adverse event connected to the therapy, as reported in a corporate statement.In order to evaluate the effectiveness of NGN-401 across a larger population, Neurogene also started a new cohort within the study including teenage and adult participants aged 16 and above. Three people are likely to be 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