David Liu has a new big idea: pro teome edit ing. It could one day shred Tau, RAS and some of the worst dis ease-caus ing ...


2021-03-04 21:02:27 ENDPOINTS NEWS


Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another. Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans. The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights. Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates. Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now. Let’s start with the latest data from Lilly. Despite all the excitement over the possibility of T cell therapies to crack the code against solid tumors, early safety data have limited the burgeoning field’s promise. A clutch of players hope to solve that problem by “masking” their drugs, and now one of those outfits has snared fresh investor cash to take its shot in the clinic. Amunix closed a $117 million Series B to guide AMX-818, a masked protease-activated T cell engager for HER2 expressing tumors, to the clinic as well as shepherding its bustling pipeline of tumor hunters through the preclinical stage, the company said Thursday. The EMA has started a rolling review of Sputnik V, the Russian vaccine that holds the dubious title of the world’s first registered jab for Covid-19. Seven months after the controversial clearance in Russia, Europe’s human medicines committee says it’s convinced to start looking at the application by data indicating that the adenovirus-based vaccine triggers the production of antibodies and immune cells against the SARS-CoV-2 coronavirus. The jury’s still out on whether the first wave of AI companies can significantly change drug development, but investors are increasingly buying into the hype. Exscientia, the decade-old UK machine learning outfit, announced Thursday that they’ve expanded their Series C, first announced in May, from $60 million to $100 million. The expansion most notably includes BlackRock, the private equity firm that has been wading deeper and deeper into biotech. They now join Novo Holdings, Bristol Myers Squibb and others among the company’s most recent backers. It’s clear by now that biopharma experienced a massive boom in 2020, but a new report out Thursday says the Massachusetts hub was particularly successful. The trade group MassBio released its latest industry snapshot, summarizing the last calendar year as the most successful for the Massachusetts biopharma sector. Overall, Massachusetts-based biotechs raised $5.8 billion in 2020, marking a hefty 93% increase from the previous year. Gilead is chopping 179 jobs in its home state of California as it scales down its headquarters in favor of a hub in North Carolina. Up to half of the roles would shift to Research Triangle Park, where the company is setting up a new business services and information technology center, the San Francisco Chronicle reported. The precise number will depend on how many employees choose to relocate. Per a WARN notice filed with the state, the layoffs are expected to be effective May 30. GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan. The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday. A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement. The FDA statement amounts to a straight slapdown, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.
在大卫·刘因发明基因编辑的新形式而出名之前,他在学术界的知名度在一定程度上是因为他的一项更默默无闻的创新:一种鲁伯·戈德堡式的系统,利用细菌感染的病毒获取一种蛋白质并将其转化为另一种蛋白质。 自2011年以来,刘的实验室使用了这套名为PACE的系统,梦想出梦幻般的新蛋白质:DNA碱基编辑器比原来的功能强大得多;基因编辑器Cas9的更多功能形式;杀灭抗药性虫子的杀虫剂;将合成氨基酸滑入生物体内的酶。但他们一直在努力掌握生物界最常见、最强大的蛋白质之一:蛋白酶,这是一组瑞士军刀酶,可以切割、裂解或撕碎从病毒到人类的任何事物中的其他蛋白质。 这场全球大流行可能扰乱了经济,夺走了数十万人的生命,扼杀了整个行业,但它对生物制药风险投资的唯一影响是帮助该领域达到令人眩晕的新高度。 根据DealForma首席执行官Chris Dokomajilar的记录,以下是业内前100名风险投资家的排名,根据他们公开参与的交易数量排名。然后,数字大师计算出他们投入每笔交易的估计金额--按玩家人数分配现金--以表明他们是如何管理自己的辛迪加的。 礼来并不只是在寻找一种更好的糖尿病药物替西帕肽。他们还想在肥胖方面超越诺和诺德的劲敌奥赞比克(赛马鲁肽)。但是,新宣称的在第三阶段关于减少A1C同时减磅的正面对决中获胜--有明显的证据表明它优于已批准的竞争对手--可能会证明现在很难卖出。 先从礼来的最新数据说起。 尽管人们对T细胞疗法破解实体瘤密码的可能性感到兴奋,但早期的安全性数据限制了这一新兴领域的前景。一批玩家希望通过“掩盖”他们的药物来解决这个问题,而现在其中一家公司已经获得了新的投资者资金,让他们在诊所里出手。 Amunix公司周四表示,该公司关闭了1.17亿美元的B系列产品,以引导AMX-818进入临床,并将其熙熙攘攘的肿瘤猎人输送到临床前阶段。AMX-818是一种用于表达HER2的肿瘤的由蛋白酶激活的T细胞参与剂。 EMA已经开始对俄罗斯的Sputnik V疫苗进行滚动审查,该疫苗被称为世界上首个注册的新冠疫苗。 在俄罗斯获得这一有争议的批准7个月后,欧洲人类药物委员会表示,有数据表明,以腺病毒为基础的疫苗会引发针对新型冠状病毒冠状病毒的抗体和免疫细胞的产生,因此该委员会确信将开始研究这一申请。 人工智能公司的第一波浪潮能否显著改变药物开发,目前还没有定论,但投资者越来越多地买进了这场炒作。 拥有十年历史的英国机器学习公司Exscientia周四宣布,他们将5月首次宣布的C系列产品的规模从6000万美元扩大到1亿美元。最引人注目的扩张包括私人股本公司贝莱德,该公司在生物技术领域越陷越深。他们现在加入了Novo Holdings,Bristol Myers Squibb和其他公司的最新支持者行列。 现在很明显,生物制药在2020年经历了一个巨大的繁荣,但周四发布的一份新报告说,马萨诸塞州的中心尤其成功。 贸易组织MassBio发布了最新的行业快照,总结了过去一年是马萨诸塞州生物制药行业最成功的一年。总体而言,总部位于马萨诸塞州的biotechs在2020年筹集了58亿美元,比上年大幅增长93%。 Gilead在其总部所在地加利福尼亚州裁减了179个工作岗位,并将总部设在北卡罗来纳州。 据《旧金山纪事报》报道,多达一半的职位将转移到Research Triangle Park,该公司正在那里建立一个新的商业服务和信息技术中心。确切的数字将取决于有多少员工选择搬迁。 根据向该州提交的警告通知,裁员预计将于5月30日生效。 葛兰素史克公司和Vir Biotechnology公司在接到NIH一项针对住院Covid-19患者的重大研究的邀请后,希望他们合作的抗体之一能够赢得胜利。但就像礼来一样,这对组合的药物不能击中目标,现在他们将被留在认真看比赛计划。 合作伙伴周三表示,NIH已经停止了GSK和Vir的抗体VIR-7831在其晚期Active-3试验中的注册,因为该药物在实现住院的Covid-19患者的持续康复方面显示出可忽略不计的效果。 一周多一点后,BrainStorm公司承认FDA的监管机构已经通知他们,该生物技术公司需要更多的数据才能获得ALS治疗NurOwn的批准--同时仍在吹捧疗效的“明确信号”,并不排除申请--该机构决定在一份最不寻常的声明中澄清记录。 FDA的声明相当于一个直接的打击,提供了一套不同于该公司去年11月公开发表的药效数据,并排除了任何统计意义的可能性。