圖片來源：123RF
1. 17億美元合作，打造治療嚴(yán)重神經(jīng)疾病的基因療法
1月29日，Neurocrine Biosciences和Voyager Therapeutics公司聯(lián)合宣布達(dá)成戰(zhàn)略合作，共同開發(fā)和推廣Voyager公司的基因療法項(xiàng)目，其中包括治療帕金森?。≒arkinson’s disease, PD）的主打在研療法VY-AADC和治療弗里德賴希共濟(jì)失調(diào)（Friedreich’s ataxia）的VY-FXN01。這一合作將Neurocrine在神經(jīng)科學(xué)和藥物研發(fā)及推廣方面的專長，與Voyager公司的創(chuàng)新基因療法項(xiàng)目相結(jié)合，開發(fā)靶向嚴(yán)重神經(jīng)疾病的新療法。
2. 開發(fā)治療大腦疾病的基因療法，SIRION與Denali達(dá)成合作
1月10日，SIRION Biotech與Denali Therapeutics宣布，兩家公司簽訂了一項(xiàng)研發(fā)許可和合作協(xié)議，SIRION將為Denali開發(fā)腺相關(guān)病毒載體（AAV），讓基因療法可以跨越血腦屏障，應(yīng)用于治療帕金森病（PD）、阿茲海默?。ˋD）、肌萎縮性側(cè)索硬化癥（ALS）、及其它神經(jīng)退行性疾病。SIRION擁有全球領(lǐng)先的以病毒為基礎(chǔ)的基因遞送技術(shù)，而Denali Therapeutics則是專注于治療神經(jīng)退行性疾病的生物技術(shù)新銳。依據(jù)協(xié)議，Denali將支付給SIRION預(yù)付款，未來里程碑付款等一系列款項(xiàng)。
3. Sangamo宣布體內(nèi)基因組編輯治療B型血友病的1/2期臨床試驗(yàn)完成首例患者治療
日前，Sangamo Therapeutics宣布，第一例患者已經(jīng)接受了1/2期臨床試驗(yàn)的治療方案。該試驗(yàn)旨在評估SB-FIX，一種用于治療B型血友病患者的體內(nèi)基因組編輯試驗(yàn)療法的療效和安全性。與傳統(tǒng)的AAV基因療法和以慢病毒或逆轉(zhuǎn)錄病毒為基礎(chǔ)的隨機(jī)插入基因組的基因療法不同，SB-FIX的設(shè)計(jì)目標(biāo)是永久和精確地將編碼凝血因子IX的基因整合到DNA中。
4. GenSight的基因療法在治療Leber遺傳性視神經(jīng)病變（LHON）的3期臨床試驗(yàn)中獲得積極結(jié)果
GenSight Biologics是一家致力于發(fā)現(xiàn)和開發(fā)創(chuàng)新基因療法，以治療視網(wǎng)膜神經(jīng)退行性疾病和中樞神經(jīng)系統(tǒng)疾病的生物醫(yī)藥公司。該公司進(jìn)行的名為REVERSE的3期臨床試驗(yàn)旨在評價(jià)玻璃體內(nèi)單次注射GS010（rAAV2/2-ND4）治療11778-ND4 Leber遺傳性視神經(jīng)病變（LHON）患者的安全性和有效性。日前，該公司宣布，第72周的3期臨床試驗(yàn)的數(shù)據(jù)分析顯示，接受基因療法GS010治療的患者與視力相關(guān)的生活質(zhì)量綜合指數(shù)得到持續(xù)改善。
5. 攜手NIH治療神經(jīng)系統(tǒng)疾病，Axovant擴(kuò)大基因療法管線
Axovant Sciences公司宣布，該公司從馬薩諸塞大學(xué)（UMass）醫(yī)學(xué)院獲得兩項(xiàng)創(chuàng)新基因療法項(xiàng)目（AXO-AAV-GM1和AXO-AAV-GM2）的全球獨(dú)家研發(fā)許可。這兩項(xiàng)基因療法分別用于治療GM1神經(jīng)節(jié)苷脂病和GM2神經(jīng)節(jié)苷脂病（又名Tay-Sachs和Sandhoff?。?。檢驗(yàn)AXO-AAV-GM1基因療法的研究人員發(fā)起的臨床試驗(yàn)將在NIH進(jìn)行。
參考資料：
[1] Neurocrine Biosciences and Voyager Therapeutics Form Strategic Development and Commercialization Collaboration for Parkinson’s Disease and Friedreich’s Ataxia. Retrieved January 29, 2019, from https://globenewswire.com/news-release/2019/01/29/1706690/0/en/Neurocrine-Biosciences-and-Voyager-Therapeutics-Form-Strategic-Development-and-Commercialization-Collaboration-for-Parkinson-s-Disease-and-Friedreich-s-Ataxia.html
[2] IRION Biotech and Denali Therapeutics Join Forces to Develop Gene Therapies for Diseases of the Central Nervous System. Retrieved Jan，10, 2019, from https://www.businesswire.com/news/home/20190109005078/en/SIRION-Biotech-Denali-Therapeutics-Join-Forces-Develop
[3] Sangamo Announces Upcoming Clinical Data Presentations At WORLDSymposium 2019. Retrieved Jan 04, 2019, from https://www.prnewswire.com/news-releases/sangamo-announces-upcoming-clinical-data-presentations-at-worldsymposium-2019-300772883.html
[4] Benitec Provides Update on BB-401 Cancer Treatment Program. Retrieved Dec 21, 2018, from https://www.biospace.com/article/benitec-provides-update-on-bb-401-cancer-treatment-program/?s=89
[5] Sangamo Announces Treatment Of First Patient In Phase 1/2 Clinical Trial Of In Vivo Genome Editing Therapy For Hemophilia B. Retrieved Dec 17, 2018, from https://www.prnewswire.com/news-releases/sangamo-announces-treatment-of-first-patient-in-phase-12-clinical-trial-of-in-vivo-genome-editing-therapy-for-hemophilia-b-300767281.html
[6] GenSight Biologics Reports Sustained Quality of Life Improvements at Week 72 of Phase III REVERSE Clinical Trial of GS010 for the Treatment of Leber Hereditary Optic Neuropathy (LHON). Retrieved Dec 12, 2018, from https://www.biospace.com/article/releases/gensight-biologics-reports-sustained-quality-of-life-improvements-at-week-72-of-phase-iii-reverse-clinical-trial-of-gs010-for-the-treatment-of-leber-hereditary-optic-neuropathy-lhon-/?s=89
[7] AXOVANT LICENSES INVESTIGATIONAL GENE THERAPIES FOR GM1 GANGLIOSIDOSIS, TAY-SACHS AND SANDHOFF DISEASES FROM UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL. Retrieved December 14, 2018, from http://investors.axovant.com/news-releases/news-release-details/axovant-licenses-investigational-gene-therapies-gm1
[8] Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1. Retrieved December 4, 2018, from https://www.novartis.com/news/media-releases/novartis-announces-fda-filing-acceptance-and-priority-review-avxs-101-one-time-treatment-designed-address-genetic-root-cause-sma-type-1
基因編輯和基因療法系列：
