第339集 - 生物科技并购、体内CAR-T疗法与FDA动荡

在癌症、肥胖和心血管领域交易的推动下，生物科技行业十亿美元以上的并购案正呈上升趋势。

Fueled by cancer, obesity, and cardiovascular deals, billion dollar plus takeouts in biotech are on the rise.

我们将在最新一期的《BioCentury本周》播客中进行深入探讨。

We'll take a closer look on the latest BioCentury This Week podcast.

此外，欢迎来到ASH星期一。

Plus, welcome to ASH Monday.

血液学学会年会上，来自奥兰多的数据正源源不断地涌现。

Data have been pouring out of Orlando from the Hematology Society's annual meeting.

我们将重点关注来自科洛尼亚的首个人体体内CAR-T细胞治疗数据。

We'll zero in on first in human in vivo CAR T data from Colonia.

还有更多数据——Praxis公司的又一次胜利为罕见遗传性癫痫治疗带来了亟需的进展。

And more data, another Praxis win provides much needed progress in rare genetic epilepsies.

Selena终于获得了她期待的数据——司美格鲁肽的完整数据集已在CTAD会议上公布。

And Selena got that data she was looking for, the full dataset for semaglutide presented at CTAD.

司美格鲁肽本应能抑制中枢神经系统炎症。

Semaglutide was supposed to quench CNS inflammation.

它没有。

It didn't.

赛琳娜将为你深入剖析细节。

Selena will dig into the details for you.

FDA又度过了动荡的一周，史蒂夫将在播客后半部分进行分析。

And another tumultuous week at FDA, Steve will give his analysis on the back half of the podcast.

我是杰夫·克兰默，BioSentry的执行编辑之一。

I'm Jeff Cranmer, one of the executive editors here at BioSentry.

今天与我同行的还有我的同事们。

And joining me today are my colleagues.

西蒙·菲什伯恩，主编。

Simon Fischburn, Editor in Chief.

赛琳娜·科赫，执行编辑。

Selena Koch, Executive Editor.

劳伦·马茨，生物制药情报执行总监。

Lauren Martz, Executive Director of Biopharma Intelligence.

还有史蒂夫·奥斯汀，华盛顿编辑。

And Steve Ostin, Washington Editor.

它回来了。

It's back.

《生物世纪》不可或缺的摩根大通指南已重新上线。

BioCentury's indispensable JPMorgan guide has returned.

访问jpmguide.com获取旧金山年度生物技术启动期间举办的场边活动和招待会内幕消息。

Visit jpmguide.com for the inside track on sideline events and receptions happening during the annual biotech kickoff in San Francisco.

嘿，如果您正在举办特别活动，请使用网站上提供的在线表格确保您的活动出现在我们的指南中。

And hey, if you're hosting something special, use the online form that you'll find there to make sure your event appears in our guide.

再次提醒，请访问jpmguide.com充分利用您的2026年摩根大通周。

Again, that's jpmguide.com to make the most of your JPM twenty twenty six week.

今天有几家因数据表现亮眼的公司股价大幅波动。

There's a couple of big movers on data today.

结构化治疗公司的股价翻倍，因其每日一次口服疗法实现11%减重效果；Chimera公司股价上涨50%，因其STAT6降解剂在中重度特应性皮炎治疗中展现出强劲的生物标志物结果。

Structured Therapeutics, obesity companies shares doubling in value as its once daily oral therapy leads to 11% weight loss and Chimera rises 50% on strong biomarker results for its STAT6 degrader for moderate to severe atopic dermatitis.

两家公司的市值都增加了数十亿美元。

Both companies gaining billions in market caps.

Steven Hansen将在biosentry.com上对Structured的数据进行分析。

Steven Hansen will have analysis of structures data on biosentry.com.

那么，我们开始吧。

So, hey, let's kick things off.

距离年底还有一个月，今年已经与过去十年中任何一年的大型并购交易数量持平。

With a month remaining, this year is already tied for the most billion dollar plus m and a deals of any year in the past ten.

Selena，这一趋势背后的原因是什么？

Selena, what's behind the trend?

是的。

Yeah.

嗯，并购交易确实是一个亮点。

Well, m and a has certainly been a bright spot.

人们一直在引用。

People keep citing.

所以我们只是做我们在这里的工作，并想量化这一点。

So we just do what we do here and wanted to quantify that.

所以到11月为止，还不是全年数据，可能还会有更多。

So by the November, so it's not yet full year data, there could be more.

今年宣布的并购交易额超过370亿美元，与2021年并列成为过去十年中规模最大的年份。

There were $37,000,000,000 plus m and a deals announced this year, time 2025 with 2021 for the biggest year in the last decade.

于是我们从几个方面进行了分析。

And so we broke it down a couple ways.

我们按疾病领域进行了分类，也按被收购公司主导产品的研发阶段进行了分类。

We broke it down by disease area, and we broke it down by the stage of the lead product of the company being acquired.

这应该不会让人感到意外，拥有上市产品的公司在这370亿交易中占了最大份额。

And it probably won't surprise anybody that, you know, companies with marketed products are the biggest set within that '37, 12.

但更有趣的是，今年有6家处于一期临床阶段的公司以10亿美元或更高的价格被收购。

But more interestingly, maybe there were six takeouts this year of a billion dollars or more for companies in phase one.

这比去年的2家有所增加，前年是1家，2016、2017年则是零，甚至还有3家临床前阶段的公司以高价被收购。

That's up from two last year, one the year before, zero in 2016, 2017, and even three preclinical companies were taken out for a high price tag.

林赛的分析与我们同事保罗·博纳诺斯撰写的一篇观点文章不谋而合，他注意到几起这类早期公司被高价收购的案例，并提出了一个问题：随着药企专利悬崖逼近且成熟资产数量有限，我们是否会开始看到收购方为早期公司支付更高溢价？

And this sort of Lindsay's analysis coincided with a perspective piece written by our colleague, Paul Bonanos, who had noticed a couple of these, you know, big money takeouts of early stage companies and had just posed the question, you know, with pharma patent cliffs looming and a limited number of, say, mature assets available for m and a, you know, are we gonna start seeing buyers pay more for early stage companies?

而林赛的数据表明，这种情况可能已经开始发生了。

And, you know, Lindsay's data suggests that maybe that's already starting to happen.

是的，我认为这非常重要，因为当人们谈论并购时，焦点往往都集中在大型企业的整合上。

Yeah, I mean, I think it's really important because a lot of the focus when people talk about M and A is consolidation of the big companies.

我们几年来一直在讨论这类十亿美元级交易的兴起，许多公司和资产的价值都集中在10亿至50亿美元这个区间。

And we've been talking for a couple of years about looking at this rise of sort of billion dollar plus deals where there's a lot of value in the, let's say, 1,000,000,000 to $5,000,000,000 range in a lot of companies and assets.

Salina，你刚才提到的内容也与专利悬崖有关。

And what you're talking about, Salina, is also regarding the patent cliff.

我们都知道很多后期资产已经被抢购一空，对吧？

Well, we know that a lot of the later stage assets have been picked off, right?

所以企业现在需要在价值链上再往前一步。

So companies now need to go a little bit earlier in the value chain.

他们仍然愿意为这些明显风险更大的赌注投入相当可观的筹码。

And they're still willing to put considerable weight behind some of those bets that are obviously riskier.

显然，强生以超过30亿美元收购了处于临床前阶段的Alda公司。

Obviously, we've got the J and J takeout of Alda for over $3,000,000,000 which is a preclinical company.

我认为这引起了广泛关注。

I think that raised a lot of eyebrows.

但我想也许你可以谈谈林赛文章中提到的治疗领域细分情况。

But I think also maybe you could talk a little bit about the breakdown of therapeutic area that Lindsay's piece laid out.

是的。

Yeah.

癌症领域依然占据主导地位。

Well, cancer remains king.

如果你看过去十年这些大型并购交易的数据，趋势线就是持续上升，2025年达到十年峰值，共有20笔交易。

If you look at the last decade's worth of these really large MA deals, it's just, the slope is just upward and to the right, with 2025 being the decade's highest number, at 20 of them.

尽管免疫炎症领域热度很高，但过去三年大型收购案数量一直稳定在7到10笔左右。

For all the hotness that has been I and I, the last three years have been really steady for these large takeouts at like seven to 10.

另一个增长领域当然是肥胖和心血管方向，最近几年呈现逐年增长态势。

And then the other growth area, of course, obesity and cardiovascular, seeing sort of year over year increases recently.

是的，显然对于公司，尤其是它们的投资者来说，鉴于IPO市场仍然非常受限，这是一条不错的退出路径。

Yeah, well, obviously for companies, and in particular their investors, this is a good exit path given that the IPO market is still very constrained.

所以我猜我们将会看到10亿美元以上的并购交易可能还会持续到2026年一段时间。

So I guess we're going to imagine the beat of $1,000,000,000 plus m and a deals is probably going to carry on for a little while into 2026.

好的，没错。

All right, yeah.

正如塞琳娜早些时候所说，这个数字是今年迄今为止90亿美元以上的并购交易，涉及那些在交易宣布时主要项目还处于临床前测试或一期临床试验阶段的公司，这是十年来最多的。

And that number, as Selena had said earlier, that's $9,000,000,000 plus M and A deals year to date for companies whose lead programs were in preclinical testing or phase one at the time the deal was announced, and that to the most in ten years.

好的，Ash。

Okay, Ash.

不可避免的。

Unavoidable.

如果你有电子邮箱，我相信你肯定收到过不少Ash的邮件。

If you have an email inbox, I'm sure a lot of Ash emails have landed in your lap.

本周，Ash将公布更多首次人体内CAR-T数据，再次展示该模式的潜力并证明其不断增长的研发管线是合理的。

This week, Ash will deliver more first in human in vivo CAR T data that once again showcases the promise of the modality and justifies the growing pipeline.

科洛尼亚治疗公司即将公布新数据。

There's new data coming from Colonia Therapeutics.

数据显示又有三名患者在接受体内CAR-T细胞疗法后达到微小残留病灶阴性。

They show that three more patients have achieved MRD negativity after treatment with an in vivo CAR T cell therapy.

我的同事劳伦告诉我，当前的问题包括：近乎完美的记录能保持多久，哪些技术将被证明最安全有效，以及这些结果如何转化应用于自身免疫性疾病？

My colleague Lauren tells me that the questions now include how long can the near perfect track record last, which technologies will prove safest and most effective, and how will these results translate to autoimmune diseases?

现在有请劳伦。

Let's bring in Lauren now.

劳伦，你将回答所有这些问题。

Lauren, you're going to answer all those questions.

谢谢，杰夫。

Thanks, Jeff.

我当然没有全部答案，但随着每个临床数据的公布，我认为我们确实离真相更近了一步。

I certainly don't have all of those answers, but with each clinical readout, I do think we get a little bit closer.

正如你提到的，从最新体内CAR-T临床数据中我们了解到：科洛尼亚的KLN101010——一种靶向BCMA的慢病毒载体体内CAR-T细胞疗法——使该研究中所有三名经过深度治疗的多发性骨髓瘤患者在治疗一个月后均达到微小残留病灶阴性。

As you mentioned, at a high level, what we've learned from the latest clinical readout for in vivo CAR T is that Kolonia's KLN101010, a lentiviral vector in vivo CAR T cell therapy targeting BCMA is that it led to MRD negativity one month after treatment for all three heavily pretreated multiple myeloma patients evaluable in that study.

我们还从摘要中了解到，至少有一位患者在三个月后仍保持MRD阴性，明天完整数据公布时我们应该能了解更多信息。

We also know from the abstract that at least one of the patients remains MRD negative at three months and we should learn more tomorrow when the full data are presented.

我认为这次数据公布的另一个重要之处在于，它被列为ASH会议的压轴报告，这充分说明了目前体内CAR-T细胞疗法引发的巨大关注。

I think another important thing about this readout is that this was profiled as a late breaker at ASH, which says a lot about how much excitement exists around in vivo CAR T cell therapies right now.

明天完整数据报告后我们将发布相关报道。

We'll have a story tomorrow once the full data are reported.

为此，我一直在研究体内CAR-T细胞疗法的研发管线。

And for that, I've been looking into the pipeline of in vivo CAR T cell therapies.

目前，我们已经找到了近40个研发项目，这是BioCentury能够确认的数量。

And right now, you know, we were able to find nearly 40 programs and this is what BioCentury has been able to identify.

我认为这充分反映了过去几年该领域取得的巨大发展。

And that I think just reflects a ton of growth that's happened over the last few years.

那么劳伦，纵观整个研发管线，有哪些

So Lauren, looking across the pipeline, what are the

其他已公布数据的项目，特别是我想问，关于疗效持久性的数据？

other ones that have reported data and importantly, I guess, durability data?

是的，Selena，我认为持久性是最大的问题，我不确定我们有多少数据来支持这一点。

Yeah, Selena, think durability is the biggest question, and I'm not sure how much data we have to support that yet.

当阿斯利康收购Eso Biotech时，我们知道他们有来自四位患者的疗效数据。

When AstraZeneca bought Eso Biotech, we knew that they had efficacy data from four patients.

我认为关于效果持久性的信息并不多。

I don't think there's much information about how durable the effects are.

而在那个案例中，同样采用的是靶向BCMA的慢病毒载体。

And in that case, again, it was a lentivirus targeting BCMA.

我认为这仍然是一个悬而未决的重大问题，这将有助于——我认为这将对这项技术最终的应用领域产生重大影响。

I think that just remains a big unanswered question and that will help, you know, I think that will have a big impact on where this technology ultimately ends up being used as well.

所以，在治疗癌症时，持久性似乎非常重要。

So, durability seems to be very important when you're treating cancers.

对于体内CAR-T细胞的自身免疫应用来说，持久性可能不那么重要。

It may not be as important for autoimmune applications of in vivo CAR Ts.

而自身免疫应用方面的研究进展稍显滞后，但我想我们也开始看到一些早期的临床概念验证了。

And the autoimmune applications are a bit farther behind, but I think we are starting to see some of the very early clinical proof of concept in those as well.

好的，谢谢你，劳伦。

Okay, thank you for that Lauren.

期待看到那个结果。

Looking forward to seeing that one.

另外两项数据结果，正如我在开头提到的，Praxis和诺和诺德关于司美格鲁肽治疗阿尔茨海默症的数据。

Two other readouts, as I mentioned at the top, Praxis and Novo's Alzheimer's data for semaglutide.

我们先从那里开始。

Let's start there.

上周的播客中已经讨论过这些数据，在阿尔茨海默病临床试验会议上再次提到了这些结果。

Discussed the readout on last week's podcast data again at the Clinical Trials on Alzheimer Disease meeting.

塞莱娜，你一直很期待看到完整数据。

Selena, you were eager to see the full data.

你了解到了什么？

What did you learn?

是的，在美国感恩节假期前几天，我们刚刚得知司美格鲁肽没有延缓病情进展，但我们没有看到任何结果或生物标志物数据。

Yeah, so the days leading up to The US holiday of Thanksgiving, just learned that semaglutide did not slow progression, but we didn't see any of the outcomes or the biomarkers.

就完整数据集而言，诺和诺德在CTAD会议前两周就掌握了这些数据，所以这只是初步结果。

So this, in terms of a full dataset, I mean, Novo had the data for like two weeks before the CTAD meeting, so it's top line.

他们并未进行大量亚组分析之类的工作，但展示了各种临床量表——主要终点CDR Sum of Box是阿尔茨海默病的标准评估工具，此外还有一系列其他标准临床功能评定量表。

They didn't do a bunch of subgroup analyses or anything like that, but they showed the, the various clinical scales, the primary endpoint, CDR Sum of Box is pretty standard for Alzheimer's, and then there's a bunch of other standard clinical functional rating scales.

最引人注目的可能是治疗组与安慰剂组随时间变化的曲线几乎完全重合。

And probably the most striking thing is that the curves over time of the treatment group and the placebo group are really just exactly on top of each other.

完全看不出任何显示治疗获益趋势的迹象。

There's no argument for a trend toward benefit or anything like that, really.

这是一次非常明确的失败。

It was a very clear failure.

虽然我们不愿看到失败（因为这对患者不利），但至少数据在这方面是清晰的。

And as much as we hate to see failures, because they're not good for patients, at least the data were clear in that regard.

但关于作用机制假说——我们总要自问：药物是否实现了预期作用？

The hypothesis though, right, so you always have to ask yourself, okay, but did the drug do what it was supposed to do?

原本假想司美格鲁肽能通过减少外周炎症来降低神经炎症，从而成为帮助患者的治疗机制。

And the hypothesis was that semaglutide was gonna decrease peripheral inflammation, which was in turn going to decrease neuroinflammation, and that was gonna be the mechanism by which it was gonna help patients.

你需要测量所有这些指标，才能判断它是否改善了外周炎症。

You have to, you know, measure all of those things to get a sense of whether or not it ameliorated peripheral, inflammation.

他们测量了高敏C反应蛋白。

It measured high sensitivity C reactive protein.

这就是诺和诺德在心血管结局试验（CVOT）中测量的指标。

That is what measured, Novo measured in CVOT trials, it's cardiovascular outcomes trials, for example.

正如预期的那样，该指标降低了约30%。

And that was reduced about thirty percent as was expected.

但这实际上是对外周炎症表征的全部内容了。

But that was the only really, that was the extent of the characterization of peripheral inflammation.

而神经炎症标志物则是一大堆指标。

And there was just a big bucket of neuroinflammation markers.

除其中一项指标有微小变化外，其他基本都没有改变。

Apart from one small change on one of them, they were just not changed.

这就引出了我的疑问：你们是否需要测试大量患者才能确定这些关键生物标志物没有变化？

So that brings up the question that I had, which is, did you have to test a huge number of patients to learn that it didn't move the needle on these key biomarkers?

他们难道不能用，我不知道，十几个或二十个病人就得出这个结论吗？

Couldn't they have figured that out with, I don't know, a dozen patients or 20 patients?

他们完全可以用更少的病人完成这项工作。

They absolutely could have done that work with fewer patients.

是的。

Yes.

对，Selena上周确实写了这个，但我也觉得Selena他们在这方面可能样本量不足。

Right, and Selena did write that last week, but I also feel like Selena, they kind of underpowered this.

因为当你写那篇文章说他们本可以做到时，要知道Steve说的非常重要，就是如何利用你的病人资源。

Because when you wrote that piece of they could have done it, know, what Steve is talking about, very important, you know, how are using your patients?

但你指出了他们会从中学到很多。

But you pointed to the fact that they would learn a lot from this.

我其实很怀疑他们到底学到了多少，因为样本量确实不足。

And I'm sort of really wondering how much they did learn from this because it was underpowered.

或许'统计功效'这个词用得不太准确。

And maybe power is the wrong word.

样本量是

Power is the

用词不当

wrong word.

我们有大约3800名患者

We're like So 3,800 patients

用'样本量'这个词确实不准确，但这项研究的设计本身也难以获取周边信息，因为它仅采用了C反应蛋白这一非常粗略的指标，对吧？

power is the wrong word, but it was not well designed really to get at the peripheral information because it only had this one very crude marker of C reactive protein, right?

尽管他们在大脑中有多个标记物。

Even though they had multiple markers in the brain.

如果药物进入大脑，他们本可以获得大量数据，比如发现它增加了这个标记物，但没有增加那个标记物。

If it had gotten into the brain, they'd have been full of data and like, oh, it increases this marker, but not that marker.

但他们并没有非常严谨地研究药物作用于外周炎症通路的哪个部分。所以你对实验设计和最终能得出的结论怎么看？

But they didn't really do a very rigorous job of understanding which part of the peripheral inflammation pathways it So was how do you feel like in terms of the design and what you're able to walk away with?

我觉得机制研究部分虽然有很多生物标记物，但还是有些不够深入。

I feel like the mechanistic piece, even though there were a lot of biomarkers, was a little underdeveloped.

是的，我同意这个观点。

Yeah, I agree with that.

比如，如果我认为外周和中枢免疫系统之间存在某种串扰，我会想知道具体有哪些候选通路在介导这种串扰？

Like if I thought, okay, there's some crosstalk between the periphery and the central immune systems, I would wanna know what are the candidate pathways for mediating that crosstalk specifically?

而这并不是C反应蛋白能说明的，对吧？

And that's not C reactive protein, right?

那只是个通用的多功能工具，并不被认为能直接在血脑屏障界面发挥作用。

That's a general all purpose tool and not thought to act directly at that interface of the blood brain barrier.

你可能会考虑做一个更小规模、更聚焦的研究，专门测试关于这种跨血脑屏障通讯可能如何发生的具体假设——但这些确实没有做。

You might imagine doing a smaller, more focused study testing those specific hypotheses of how those, that communication might happen across the blood brain barrier, which really wasn't done.

关于外周如何影响中枢炎症的另一个设想是：血脑屏障完整性被破坏，外周免疫细胞得以进入。

Another idea for how the periphery can influence central inflammation is by the blood brain barrier integrity kind of breaking down and peripheral immune cells getting in.

他们确实有两三个血脑屏障完整性的生物标志物数据，但这些指标在索马鲁肽作用下没有变化。

They did have a few, two, I think, biomarkers of blood brain barrier integrity, and those were unchanged by semaglutide.

塞莱娜，我听到的核心意思是这种分子其实没有很好地进入大脑，对吗？

Well, Selena, what I'm kind of hearing is that this molecule didn't really get into the brain very well, right?

是的，这是个悬而未决的问题。

Yeah, so that's an outstanding question.

根据临床前数据和其他现有数据来看，它可能根本无法以可观量进入大脑。

The thought is based on the preclinical data and other data that's available, that it probably doesn't get into the brain at any appreciable amount.

诺和诺德确实做过一个小型研究，是与三期试验同步进行的而非提前，该结果也在CTAD会议上展示过。

Novo did do a small study, like in conjunction with the phase three trials rather than before, which is also presented at CTAD.

在10人中有8人，在接受索马鲁肽治疗后脑脊液中检测到微量索马鲁肽，浓度大概是...我想说可能是每升0.1纳克左右。

In, in eight out of ten people, there was a small amount of semaglutide in the cerebral spinal fluid after semaglutide treatment, at, oh, I want to say maybe a tenth of a nanogram per liter, something like that.

这个浓度远低于血浆中的水平，而且奇怪的是似乎与血浆浓度没有相关性。

So far less than you're seeing in the plasma and strangely did not seem to correlate with the level in the plasma.

彼得·约翰逊在讨论该结果时特别强调：即使有微量进入脑脊液，与能否穿透进入脑实质形成有效浓度完全是两个不同的问题。

And Peter Johansson, who talked about that, said very specifically, look, even if small amounts get into CSF, it's a very different question whether or not any of that penetrates into the brain parenchyma and any appreciable

好的，现在让我回到史蒂夫的问题——或者说史蒂夫的观点：你们对3000多名患者做了这个试验。

Okay, so let me go back to Steve's question now, or Steve's point, which is you do this trial on 3,000 plus patients.

你们难道不觉得应该在药物能否入脑这个问题上做更多研究吗？

You don't think you might do a little bit more work on whether the molecule gets in the brain?

我知道，我知道。

I know, I know.

在开展近4000名患者的III期试验之前，本可以做一些前期工作，尤其是考虑到患者作为试验资源的珍贵性。

There are things that could have been done before jumping into a nearly 4,000 patient phase three trial, especially given the precious resource that patients are for trials.

好吧，让我们来看点好消息。

Well, let's jump to some good news.

Praxis精准医药公司的股价上周五上涨了31%，原因是其针对几种发育性癫痫性脑病亚型的钠通道调节剂在II/III期研究中因疗效显著而提前终止。

Shares of Praxis Precision Medicine rose 31% on Friday after a phase twothree study of the company's sodium channel modulator for a couple of subtypes of developmental epileptic encephalopathy was stopped early for efficacy.

该股今日仍在继续上涨。

Stock is still moving up today.

这些数据来自美国癫痫学会年会。

These came out of the American Epilepsy Society Annual Meeting.

完整数据已于周六公布。

The full data came out Saturday.

劳伦，这是一种小分子药物。

Lauren, this is a small molecule.

为什么这很重要？

Why is that significant?

我知道他们评估的癫痫发作与自闭症谱系障碍的高风险相关。

I know the seizures that they were evaluating are associated with a high risk of autism spectrum disorders.

你在撰写这篇报道时了解到了什么？

What did you learn in writing this story?

是的，我认为这种小分子药物正是让这些数据对Praxis乃至整个领域如此令人兴奋的原因之一。

Yeah, I think the fact that this is a small molecule is one of the things that makes this data so exciting for Praxis and just for the field.

这种发育性和癫痫性脑病特别是由SCN2A和SCN8A基因突变引起的遗传形式。

This developmental and epileptic encephalopathy is specifically a genetic form caused by mutations in SCN2A and SCN8A.

而现在我们有了可以专门治疗这种疾病遗传形式的小分子药物。

And here we have a small molecule that can treat specifically the genetic form of this disease.

要知道，今年推进细胞和基因疗法通过临床阶段特别困难。

You know, it's been a particularly difficult year to advance cell and gene therapies through the clinic.

当你观察这类发育性癫痫脑病的研发管线时，会发现很多开发都集中在细胞和基因疗法上，尤其是基因疗法。

When you look at the pipeline for these types of DEEs, a lot of the development is focused in cell and gene therapies, gene therapies in particular.

考虑到商业障碍和一些安全性问题，我认为这很好地提醒我们，随着小分子药物研发的进步，在某些情况下有机会设计出治疗这类遗传性疾病的药物。

And so just given the commercial hurdles and some of the safety issues, I think it's just a good reminder that with the advances that we're seeing in small molecule drug development, there is an opportunity in some cases to design drugs that can treat these kinds of genetic disorders.

另外我认为这里非常有趣的一点是，这其中还涉及自闭症的遗传因素。

And then the other thing that I think is really interesting here is that there's an autism, a genetic autism component involved here as well.

所以这是一项专注于这些基因突变相关癫痫的临床试验。

So this is, you know, a trial that's focused on epilepsy associated with these mutations.

比如SEN2A同时也是自闭症中最明确的遗传风险因素之一。

But SEN2A, for example, is also one of the most well characterized genetic risk factors for autism.

我认为帮助恢复钠通道功能的进展可能会对自闭症产生影响。

I think progress in helping restore function of the sodium channel could have an impact on autism.

目前由于各种原因，这绝对是药物研发的热门领域。

This is definitely a hot area for drug development for different reasons right now.

是的，在这个基因疗法融资极其困难的时代，考虑到我们今年看到的安全性问题以及持续存在的商业挑战，治疗方式的选择可能非常重要。

Yeah, and probably worth pointing out in this era of really tough funding for gene therapies, the safety issues we've seen this year, the continued commercial challenges, that modality piece is probably really important.

而且这不是首例，我们看到罗氏某种程度上证明了小分子药物可以进入反义和基因治疗领域并取得竞争优势。

And it's not the first, you know, we saw Roche kind of prove this, that a small molecule can come into a space where there's antisense and gene therapy and kind of outcompete.

那是在脊髓性肌萎缩症中的VRISD。

That was the VRISD in spinal muscular atrophy.

是的，但我认为这两件事实际上都在自闭症领域取得了切实进展。

Yeah, but I think that's, you know, both of these things are sort of converging on real actual steps within autism.

显然，这里面掺杂了很多政治因素。

Obviously, there's a lot of noise, political.

这部分内容我们将在休息后继续讨论，杰夫，关于这些政治层面的问题。

That part of the program we'll get to after the break, I guess, Jeff, in terms of the politics around these things.

但这显然与癫痫有关，而癫痫是某些自闭症谱系障碍的一个特征。

But this is obviously about epilepsy, which is a feature of some autism spectrum disorders.

但我认为人们相信这可能对自闭症的一些认知特征或其他特征也有益处。

But I think that there's a belief that there may also be benefits for some of the cognitive, let's say, features or other features of autism.

我想说的是，这总体上是好消息，不仅对癫痫部分，而且对遗传学和这些不同方法正在取得进展的理念也是如此。

And I think really what I'm trying to say is this is generally good news, not only for the epilepsy part of it, but also for the idea that with genetics and with these various approaches, there is progress being made.

也许进展缓慢，但确实在自闭症领域取得了进步。

Maybe slowly, but progress being made within autism.

你是这么看的吗？

Is that how you look at it?

是的，西蒙娜，我就是这么认为的。

Yeah, Simone, that is how I see it.

而且我认为同样重要的是要注意到，这项提前终止的INBOLD研究特别关注了癫痫发作减少作为临床疗效的终点指标。

And I think it's also important to note that this INBOLD study that was stopped early specifically looked at seizure reduction endpoints for clinical efficacy.

但Praxis精准医疗公司还在进行第二项注册研究，该研究针对DEEs（发育性癫痫性脑病）不受限的情况，不局限于这些特定基因突变。

But Praxis Precision Medicine is also running a second registrational study that is looking at DEEs unrestricted, you know, not limited to these specific mutations.

在那项研究中，他们更广泛地考察了不同的疗效终点指标。

And in that study, they are looking at different efficacy endpoints more broadly.

其中一些指标可能与自闭症谱系障碍其他方面的症状更相关。

Some of those are related to symptoms that may be more relevant to other aspects of autism spectrum disorders.

我认为这将是关键所在。

I think that's going be key.

另外一点是，如果治疗介入得非常非常早，对吧？

And the other thing is if you treat very, very early, right?

这些基因缺陷本身就携带着发展自闭症的风险。

So these carry with them these genetic defects risk of developing autism.

所以我好奇我们是否有一天能在真实世界数据中看到这种风险的降低。

So I wonder if we would see, you know, in the real world data someday, a decrease in that risk.

好的，我们将进入一个短暂的休息时间。

All right, we're going to cut to a quick break.

所有这些报道你都可以在biocentury.com和biocenturypodcast.com上找到。

All of those stories you'll find on biocentury.com, biocenturypodcast.com.

如果你喜欢我们的节目，请点赞并订阅本周的BioCentury。

If you like what you're hearing, like and subscribe to BioCentury this week.

稍后我们将回来与华盛顿编辑史蒂夫·休斯顿讨论华盛顿的疯狂一周。

We'll be back to talk about a wild week in Washington with our Washington editor, Steve Huston.

本周的BioSentry由韩国第五届东西方生物制药峰会赞助播出。

BioSentry This Week is brought to you by the Fifth East West Biopharma Summit in South Korea.

一条创新弧线正在亚洲形成，西方生物制药领导者们正密切关注着从跨境交易到新机遇的种种变化。

An arc of innovation is emerging across Asia, and western biopharma leaders are taking note, from cross border deals to new goes.

2026年3月，第五届BioSentry Bayhelix东西方生物制药峰会首次在韩国举行。

In March 2026, the fifth BioSentry Bayhelix East West Biopharma Summit visits South Korea for the first time.

与将韩国创新推向全球的生物制药领袖会面。

Meet the biopharma leaders putting Korea innovation on the global map.

了解韩国为何已成为临床试验和制造中心。

Learn why Korea has become a clinical trial and manufacturing hub.

探索韩国是否会成为新公司成立的下一个热点地区。

Discover if Korea is the next hotspot for new co formation.

此外还能结识来自印度、新加坡、中国和日本的生物制药创新者。

Plus meet biopharma innovators from India to Singapore to China and Japan.

立即访问biocenturyeastwest.com进行注册。

Register now at biocenturyeastwest.com.

欢迎回到《BioCentury本周要闻》播客。

We are back on the BioCentury This Week podcast.

让我们把目光转向华盛顿。

Let's get to Washington.

史蒂夫，我想你上周提交的报告数量创下了个人纪录，我们该从哪儿说起呢？

Steve, I think you had a personal best in the number of reports that you filed last week and where to begin?

嗯，我想上周我们结束播客时，理查德·帕兹德还是FDA药物评估与研究中心的主任。

Well, I think that when we left listeners to the podcast last week, Richard Pazder was still the the director of the Center for Drug Evaluation and Research at FDA.

当时人们对CBER主任维奈·普拉萨德撰写的一份关于疫苗政策和新疫苗监管框架的备忘录存在一定程度的担忧。

There was, some level of concern around a memo that Vinay Prasad, the director of CBER, had written about vaccine policy and and a new framework for vaccine regulation.

而现在情况又发生了变化，就像过山车又来了个急转弯，这次简直是个桶滚式翻转。

And things have look, we've had another another turn on the on the roller coaster, and this one is, you know, kind of a barrel roll.

首先，理查德·帕斯特宣布将于十二月开始退休。

So first, Richard Pastor announced his retirement starting at the December.

所以他在任还不到三周就离职了。

So he was on the job less than three weeks, and then he left.

几乎所有在世的前FDA专员都联名在《新英格兰医学杂志》上发表评论，警告FDA的新疫苗政策构成公共卫生危机，并对马蒂·马卡里专员管理FDA的方式表达了更广泛的担忧。

Almost every living former FDA commissioner signed on to a commentary that was published in the New England Journal of Medicine warning that FDA's new vaccine policies constitute a public health crisis and expressing broader concerns about commissioner Marty Makary's management of the agency.

随后特蕾西·贝丝·霍克被任命为代理CEDAR主任，接替理查德·帕斯特的职位——而帕斯特在未来几周将重新担任肿瘤卓越中心主任。

And then Tracy Beth Hoek was named acting CEDAR director to replace, Richard Pastor, who for the next few weeks has come back to being head of the Oncology Center of Excellence.

所以当理查德·帕斯特宣布作为高级主管退休时，你可以听到全美乃至全球生命科学高管们的倒吸凉气声。而当他们开始收到特蕾西·贝丝·霍格被任命为CEDAR代理主任的短信通知时，你又能听到电话纷纷掉地的声音。

So you could hear the gasps from life sciences executives around the country and around the world when they heard that Richard Pastor was retiring as senior director, And then you could hear the phones hitting the floor when they started getting the texts announcing that Tracy Beth Hoag had been named acting CEDAR director.

简而言之，在FDA内部和受监管行业看来，她既不胜任这个职位，又是个意识形态至上者。

So the short form, she's viewed inside FDA and among regulated industry as unqualified for the job and an ideologue.

她很可能会引发FDA的辞职潮。

She's likely to provoke a wave of resignations at FDA.

这是我从机构内部人员那里听到的消息。

That's what I'm hearing from people in the agency.

而且我认为这会给药物研发者带来更大的不确定性。

And I think greater uncertainty for drug developers.

她的任命和帕斯特的离职——或者说即将离职——我认为都表明药物评审将受到政治压力和意识形态影响。

Her appointment and Pastor's departure, or his imminent departure, I think, are signs that drug reviews are going to be influenced by political pressures and ideological influences.

在我们讨论霍格博士担任CEDAR负责人之前，稍等一下。

Just a minute before we get to Doctor.

史蒂夫，我们一直在进行一项调查，现在已经截止了，本周会公布结果。

Hoag as the head of CEDAR, Steve, we've been running a survey, which is now closed, we'll be getting that out this week.

根据调查反馈，给大家提个醒，从调查结果以及上周我们与生物制药公司和生物科技负责人在各种会议上的大量个人互动和交谈来看，人们普遍认为帕斯塔的任命是FDA最大的稳定力量。

From the survey responses, here's a heads up to everybody, from the survey responses, as well as from a lot of personal interactions and conversations that we had at various meetings last week with biopharma BCs and biotech heads, there was really a feeling that Pasta's appointment was the biggest stabilizing force for FDA.

他就像房间里那个成熟稳重的主心骨。

He was the grown up in the room kind of thing.

我认为最重要的就是稳定性。

Most of all, I think, is the stability.

现在他的离职确实让很多人对明年非常担忧，甚至在他们知道下一任CEDAR主任是谁之前就这样了，对吧？

Now, his departure has certainly got a lot of people very worried about next year, even before they knew who the next CEDAR director would be, right?

那么，史蒂夫，你有什么好消息吗？

Is there in terms of Have you got any good news, Steve?

有什么能安抚人心的好消息可以告诉大家吗？

Any upside calming things that you can tell people out there?

从FDA方面来看，没有什么好消息。

From FDA, there's no good news.

我认为有些消息可能被解读为潜在的好消息，对吧？

I think there's news that people interpret as the possibility of good news, right?

我们之前在播客中讨论过，存在这样一种合理的机制路径——暂且不论其具体名称——它有可能为治疗极罕见病症及定制疗法打开研发之门。

So, you know, we talked about it on podcast before, there's this plausible mechanism pathway, which leaving aside the name of it, has the potential to unlock the development of therapies for very rare conditions and bespoke therapies for very rare conditions.

但目前远不能确定该机制会以真正实现这一目标的方式落地执行。

But it's far from certain that it's going to be implemented in a way that actually does that.

但如果你在寻找好消息，那里确实存在可能性。

But if you're looking for good news, you know, there's a potential there.

上周还发生了另一件事：FDA内部酝酿已久的一项提案——由Marty Makary专员和CBIR主任Vinay Prasad推动发布声明，确立FDA未来的默认立场是要求企业仅需进行一项关键临床试验即可支持药物获批。

There's also something else that happened last week, which was that one of the things that's been in the works at FDA for some time is an idea that Commissioner Marty Makary and CBIR Director Vinay Prasad have of putting out a statement saying that the default assumption at FDA going forward will be that companies will have to perform a single clinical trial, single pivotal clinical trial to support drug approval.

他们长期试图通过HHS法律办公室发布该声明，但始终未能获得最终签署。

They had been trying to get a statement saying that through HHS legal office for some time and hadn't been able to get final sign off on it.

McCary专员上周向另一家媒体的记者透露并基本泄露了这份备忘录内容。

Commissioner McCary discussed it, basically leaked the memo to a reporter from another publication last week.

因此虽然媒体多有报道，但正式新闻稿仍未发布，我们尚不清楚其具体内容。

So it's been in the news quite a bit, but the the press release still hasn't been released, so we don't know exactly what's in it.

更重要的是，我们不知道FDA将如何将其付诸实践。

And what's more, we don't know how FDA might put it into practice.

所以你可以把这看作是好消息。

So you could look at that as good.

你也可以把它视为坏消息。

You could look at it as bad.

这无疑会带来极大的不确定性。

It's certainly going to create a great deal of uncertainty.

要知道，将新闻稿转化为可预测的政策将极具挑战性。

You know, translating a press release into predictable policy is going to be challenging.

好的。

All right.

那么你的预期是什么？

So what are your expectations?

关于霍格医生，我们需要了解什么？

What do we need to know about Doctor.

霍格？

Hogue?

她是一名专攻运动医学的医学博士。

So she's an MD specializing in sports medicine.

她拥有流行病学博士学位。

She has a PhD in epidemiology.

她从未主持过临床试验。

She's never run a clinical trial.

她从未参与过药物评审。

She's never reviewed a drug.

她完全没有监管机构的工作经验。

She has no regulatory experience whatsoever.

她在疫情期间以新冠异议者身份崭露头角。

She rose to prominence as a COVID contrarian during the pandemic.

她曾为佛罗里达州卫生局长约瑟夫·拉达波提供反疫苗政策建议，这些政策受到公共卫生专家的强烈批评。

She advised Florida surgeon general Joseph Ladapo on anti vaccine policies that have been heavily criticized by public health experts.

作为FDA派驻AASIP（即CDC免疫实践咨询委员会）的代表，她在该职位上质疑儿童强制接种疫苗的必要性，并发挥了领导作用。

She's played a leading role as FDA's representative to the AASIP, that's the CDC Advisory Committee on Immunization Practices, in in her capacity there, she's challenged the need for mandatory vaccination of children.

她呼吁修改疫苗接种计划，这将导致美国儿童接种的疫苗数量减少。

She's called for changes to the vaccine schedule that will result in American children receiving fewer vaccines.

作为FDA专员麦卡里的高级顾问，霍格一直密切参与专员的全国优先券计划。

As a senior advisor to FDA commissioner McCary, Hoag's been closely involved with the commissioner national priority voucher program.

她与CBR主任维迪·普拉萨德关系密切，并协助撰写了一份备忘录，即我之前提到的那份，宣布新的疫苗政策。

She's close to CBR director Videy Prasad and helped write a memo, the one that I mentioned earlier, announcing new vaccine policies.

上周，除斯蒂芬·哈恩外，所有在世的前FDA专员都签署了一篇评论文章，发表在《新英格兰医学杂志》上，谴责那份备忘录，称其将对疫苗开发和公共卫生造成灾难性后果。

Last week, every living FDA commissioner except one, Stephen Hahn, signed a commentary that was published by the New England Journal of Medicine that condemned that memo, saying it will have disastrous consequences for vaccine development and public health.

我认识的与FDA霍格有过接触的人说她具有科学好奇心。

People I know who have interacted with Hogg at FDA say she's scientifically curious.

在会议中，她会提出深入的问题或想要了解细节，但他们也表示她更像倡导者而非监管者，总是先有结论，然后挑选数据来达到预期结果。

In meetings, she asks probing questions or wants to get into the details, but they also say that she's an advocate, not a regulator, that she starts with conclusions and cherry picks data to get to the desired response.

与我交谈过的FDA工作人员表示，霍格采用了这种方法，她以科学上不恰当的方式使用观察性数据来断言药物安全性。

And the FDA staff I've spoken with say Hoag has used that that approach, and she's used observational data in ways that aren't scientifically appropriate to make assertions about drug safety.

一个例子是，她负责了一项关于COVID疫苗的审查，导致FDA长期任职的科学家和公务员提出了限制措施。

One example, she was in charge of a review of COVID vaccines that led to restrictions that were posed by longtime scientists and civil servants at FDA.

FDA疫苗部门前副主任菲尔·克劳斯和前代理FDA首席科学家卢西亚诺·博里奥在我们BioCentry上发表评论文章，谈及霍格对新冠疫苗采取的行动。

Phil Krausz, the former deputy head of vaccines at FDA, and Luciano Borio, the former acting FDA chief scientist, wrote a commentary that we published in BioCentry about the actions that Hoag took on COVID vaccines.

他们写道：基于错误科学假设而不断改变标准、在后期要求新数据的行为，已经侵蚀了信任、延误了重要工具的获取，并打击了开发人员推进疫苗创新的积极性。

And they wrote that shifting standards and late stage demands for new data based on faulty scientific assumptions have eroded trust, delayed access to important tools, and discouraged developers from advancing vaccine innovation.

我认为我们可以预见POG将推进MAHA优先事项：限制青少年获取SSRIs等精神类药物，采取措施减少青春期阻断剂的获取，支持迷幻药获取渠道，以及其他我认为属于MAHA议程的步骤。

I think we can expect that POG's gonna move on MAHA priorities, restricting access to SSRIs and other psychiatric drugs for adolescents, taking action to reduce access to puberty blockers, supporting access to psychedelics, and and other other steps I think that are, you know, on the MAHA agenda.

这就是我们预计她会采取的行动。

That's what we can expect that she's gonna do.

史蒂夫，我有点好奇。

Steve, I'm just curious.

比尔·卡西迪对FDA近期发生的事发表过评论吗？

Has Bill Cassidy made any comments about what's been happening at FDA?

你指的是参议院卫生教育劳工委员会主席卡西迪参议员吧。

So you're referring to senator Cassidy who's the chairman of the Senate Health Education Labor Committee.

他确实就ASIP发表过公开声明。

He has made public statements about the ASIP.

他对ACIP在减少新生儿接种乙肝疫苗机会方面所做的决定感到极其愤怒。

He's really, really kind of apoplectic about what A CIP has done in terms of reducing access of newborns or recommendations for newborns to get access to hepatitis B vaccines.

这是卡西迪非常熟悉的领域。

It's a field that Cassidy knows well.

他曾是一名医生，治疗过许多乙肝患者，因此对AZIP的所作所为极为关注。

He was a he's a physician and he treated a lot of hepatitis B patients, and he's extremely concerned about what AZIP has done.

我还没听到他就FDA问题发表过公开声明。

I haven't heard him say anything publicly about FDA.

史蒂夫，我认为我们在疫苗问题上会更认同传统观点，而不是MAHA议程。

Steve, well, I think we would agree more with the orthodoxy on vaccines than, let's say, on the MAHA agenda.

我理解那位医生的立场。

And I understand that Doctor.

霍格医生对新冠疫苗发表过很多看法，同时也承认对此存在不同观点。

Hogue has sort of had a lot to say about the COVID vaccines, also acknowledging that there are many people with different views about that.

你认为她关于新冠疫苗的立场在担任CEDAR负责人后会起到多大作用？

How relevant do you think her COVID vaccine position is going to be in her role as the head of CEDAR?

这两者之间有分歧吗？

Is there daylight between those?

她在CEDAR并不负责疫苗监管。

Well, she doesn't regulate vaccines at CEDAR.

CEDAR不负责疫苗监管工作。

CEDAR doesn't regulate vaccines.

但我认为这表明了她的思维方式，以及她确实愿意并渴望基于意识形态原则而非科学共识采取行动和监管措施。

But I think that what it shows is how she thinks about things and the fact that she's willing and eager really to take actions and to take regulatory actions based on ideological principles rather than the scientific consensus.

她的行为也与马蒂·马卡里担任FDA专员期间的做法一致——在缺乏公众咨询委员会或其他能对FDA影响公共卫生、患者健康以及企业药物研发能力的决策提供透明度和问责机制的情况下行事。

She's also acting, and this is consistent with what Marty Makary has been doing as FDA commissioner, in the absence of public advisory committees or other forms that would create transparency and accountability around FDA's actions on topics that affect public health and the health of individual patients, not to mention the ability of companies to develop drugs.

好的史蒂夫，华盛顿议程上还有其他几件事。

Alright, Steve, a few other things are on the agenda in Washington.

我不清楚能完成多少。

I don't know how much is gonna get done.

我们一直在讨论《生物安全法案》的卷土重来。

We've been talking a lot about the comeback of the Biosecure Act.

这个法案目前进展如何？

Where does that stand?

《生物安全法案》已被纳入众议院版本的《国防授权法案》(NDAA)，这是一项必须通过的立法。

So, the Biosecure Act has been included in the house version of the National Defense Authorization Act, the NDAA, which is a must pass bill.

这项法案很快就会通过。

It's a bill that's gonna be passed very soon.

几乎可以肯定的是，《生物安全法案2.0》将被纳入该法案。

It's almost certain that Biosecure two point o will be included in the bill.

听众可能记得我们在播客中讨论过这个问题。

People may remember we've talked about this on the podcast.

新版本的《生物安全法案》与去年悬而未决的版本不同，它不再列出具体公司名单，而是建立了一套流程让现政府（目前是特朗普政府）来判定哪些公司属于所谓的'受关注生物技术企业'。

The new version of Biosecure differs from the version that was pending last year because rather than listing specific companies, it creates a process for the administration, right now the Trump administration, to determine whether companies are so called biotechnology companies of concern.

对于这些被认定的企业，美国公司或希望在美国销售产品的企业将受到限制，不得使用它们的服务。

And for those companies, there would be restrictions on US companies or companies who want to sell their products in The US accessing their services.

该法案设有五年豁免期，同时也限制了制裁效力——即这些制裁对相关企业在美国销售药品能力的实际影响。

There's a five year grandfathering period, and there's also limits on the effectiveness of the of the sanctions, the effect of the sanctions on the ability of companies to sell drugs in The United States.

很大程度上将取决于特朗普政府如何解释或实施这项法律。

A lot is gonna depend on how the law is interpreted or implemented by the the Trump administration.

另一项几乎肯定会被纳入《国防授权法案》的是《对抗中国法案》，该法案对某些中国实体实施制裁，并限制或要求美国对中国特定高风险技术领域的投资进行报备。

Another bill that's almost certain to be included in the NDAA is something called the Fight China Act, which imposes sanctions on certain Chinese entities and restricts or requires notification of US investments in specified high risk technologies in China.

本质上，它建立了一套对外投资审查机制。

Basically, it establishes an outbound investment screening regime.

该法案并非直接针对生物技术领域，事实上其中某些条款还为在中国开展的特定研究活动提供了豁免空间。

It isn't directly targeted at biotech, and in fact there are provisions in it that they kind of provide a carve out for certain research activities that are conducted in China.

但尽管如此，它仍可能对生物技术公司产生影响。

But nonetheless, it's likely to impact biotech companies.

同样，很大程度上将取决于特朗普政府如何解释该法案，但某些类型的合作、某些对中国企业的投资——例如使用中国自主研发的先进芯片组或中国先进人工智能系统的企业——可能需要履行报备义务，甚至根据政府解释被迫撤资在华项目。

Again, a lot of it's gonna depend on how the Trump administration interprets it, but some kinds of collaborations, some kinds of investments in companies in China that use advanced chipsets, for example, that are developed in China or use advanced AI systems that are Chinese systems could be subject to notification requirements or even have to disinvest in in projects in China depending on how the act is interpreted by the administration.

好的。

Okay.

您可以在biosentrypodcast.com上阅读史蒂夫的所有报道。

You can read all of Steve's stories, biosentrypodcast.com.

我们下周会回来。

We will be back next week.

别忘了本周订阅BioSentry，并查看我们姐妹播客《BioSentry Show》的最新内容。

Don't forget to subscribe to BioSentry this week, and check out the latest from our sister podcast, the BioSentry Show.

Selena与Bill Hinshaw坐下来讨论了精准医疗。

Selena sat down with Bill Hinshaw to talk precision medicine.

Bill是四家生物治疗公司的CEO。

Bill is CEO of four Biotherapeutics.

本周Kendall Square Orchestra为BioCentury提供了音乐。

Kendall Square Orchestra provides the music for BioCentury this week.

该团体通过音乐连接科学与技术专业人士及波士顿社区其他成员，在支持医疗保健和教育相关事业的同时，促进协作、创新与启发。

The group connects science and technology professionals and other members of the greater Boston community to collaborate, innovate, and inspire through music while supporting causes related to health care and education.