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uniQure Stock Surges 76% on FDA Breakthrough for Huntington’s Gene Therapy AMT-130

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uniQure Stock Surges 76% on FDA Breakthrough for Huntington's Gene

Shares of uniQure N.V. skyrocketed more than 75 percent Wednesday, closing in on $47.49 after the gene therapy company announced a major regulatory advancement for its experimental treatment AMT-130 targeting Huntington’s disease. The surge reflects renewed investor optimism around the potential for the first disease-modifying therapy for the devastating neurological disorder.

uniQure stock opened sharply higher and maintained strong gains throughout the morning session on the Nasdaq. The move more than doubled the company’s market capitalization in a single trading day, erasing earlier setbacks and highlighting the high-stakes nature of biotech investments tied to clinical and regulatory milestones.

The catalyst was confirmation that the U.S. Food and Drug Administration has agreed uniQure can pursue a Biologics License Application using existing Phase I/II data from the AMT-130 program, supporting a path toward accelerated approval. This development reverses prior regulatory hurdles and accelerates timelines for potential market entry.

Regulatory Progress on AMT-130

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AMT-130 is a one-time, AAV-based gene therapy designed to lower levels of the mutant huntingtin protein responsible for Huntington’s disease. The disorder affects an estimated 30,000 people in the United States with another 200,000 at risk, causing progressive motor dysfunction, cognitive decline and psychiatric symptoms with no approved disease-modifying treatments currently available.

Earlier Phase I/II data showed promising results, including a statistically significant 75 percent slowing of disease progression at 36 months in the high-dose cohort as measured by the composite Unified Huntington’s Disease Rating Scale compared to external controls. Additional functional improvements were noted across key endpoints.

The FDA’s updated position allows uniQure to submit a BLA potentially as early as the third quarter of 2026, pending final alignment. This follows patient advocacy efforts, including petitions with tens of thousands of signatures, and comes amid broader shifts in FDA leadership and priorities for rare disease therapies.

Company executives expressed confidence in the data package. The therapy uses a precision delivery approach directly into the brain, aiming to provide long-lasting benefits from a single administration.

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Company Background and Pipeline

uniQure, headquartered in the Netherlands with significant U.S. operations, specializes in AAV gene therapies for rare and severe genetic diseases. Its platform has delivered approved therapies in hemophilia B and other areas, providing foundational experience for the Huntington’s program.

Beyond AMT-130, the company is advancing candidates in Fabry disease and other indications. Recent updates on AMT-191 for Fabry showed sustained enzyme activity improvements and patients discontinuing enzyme replacement therapy.

Financially, uniQure has faced typical biotech pressures with ongoing research and development costs. First-quarter 2026 results showed a net loss, but the regulatory clarity could open doors to partnerships, additional funding or commercialization revenue if approved.

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Market Reaction and Analyst Views

The dramatic share price increase reflects the binary nature of biotech catalysts. Prior to Wednesday’s news, the stock had traded in a range influenced by earlier regulatory uncertainty and broader sector volatility. Analyst price targets vary widely, with some forecasting substantial upside if AMT-130 reaches the market.

Wall Street has generally maintained a positive stance on uniQure’s potential, citing the unmet need in Huntington’s and the strength of the clinical data. However, risks remain, including manufacturing scale-up, long-term safety monitoring and competition from other approaches in the gene therapy space.

Trading volume spiked significantly as retail and institutional investors reacted. The stock’s movement also lifted related names in the gene therapy and neurological disease sectors.

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Broader Implications for Gene Therapy Field

Wednesday’s announcement underscores evolving FDA flexibility for serious rare diseases with strong surrogate or early clinical signals. Huntington’s represents a particularly challenging area due to its genetic basis and progressive nature, making any meaningful slowing of decline highly impactful.

Patient advocacy groups welcomed the news. Organizations like Help4HD have long pushed for accelerated pathways, viewing AMT-130 as a potential game-changer for families affected by the hereditary condition.

The development arrives as the gene therapy sector matures, with more products gaining approvals and real-world evidence accumulating. Challenges around cost, access and delivery methods persist, but successes like uniQure’s could encourage further investment.

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Risks and Considerations

Despite the enthusiasm, hurdles remain before any potential approval. Full data review, manufacturing inspections and possibly additional confirmatory studies could influence timelines. Pricing and reimbursement discussions for one-time therapies often prove complex given the high upfront costs.

uniQure will need to demonstrate consistent safety and efficacy at commercial scale. Long-term follow-up data will be critical, as gene therapies can produce effects that evolve over years.

For investors, the volatility inherent in clinical-stage biotech remains pronounced. While today’s surge rewards risk-takers, future developments around clinical holds, competitive data or macroeconomic factors could drive sharp reversals.

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Looking Ahead

uniQure plans further engagement with regulators and expects to provide additional updates on the BLA process in coming months. Positive momentum could also support partnership discussions or capital raises to fund commercialization preparations.

The Huntington’s community awaits more details, with hope that AMT-130 could transform care for a disease that has long lacked meaningful interventions. As the company advances toward potential approval, attention will turn to execution and the therapy’s real-world impact.

Wednesday’s trading action caps a period of anticipation for uniQure and highlights the sector’s capacity for rapid value shifts on regulatory news. As the gene therapy landscape evolves, uniQure’s progress with AMT-130 positions it as a key player in addressing one of medicine’s most challenging genetic disorders.

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Politics And The Markets 07/27/26

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OneWater Marine Inc. (ONEW) Q1 2026 Earnings Call Transcript

This is the forum for daily political discussion on Seeking Alpha. A new version is published every market day.

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The comments below are not regulated with the same rigor as the rest of the site, and this is an ‘enter at your own risk’ area as discussion can get very heated. If you can’t stand the heat… you know what they say…

More on Today’s Markets:

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Seeking Alpha’s Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.

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ASX 200 Slides on Trump Tariff Fears and Wall Street Selloff, Marking a Third Straight Weekly Decline

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Australia Housing Market 2026: Two-Speed Boom Persists as Prices Hit

Australia’s benchmark share index closed lower Friday, reversing gains from earlier in the week as a fresh round of U.S. tariff threats and a sharp selloff on Wall Street weighed on sentiment, capping a third consecutive weekly decline for the local market.

A rough end to the trading week

The S&P/ASX 200 fell 66.70 points, or 0.75%, to close at 8,772.30 on Friday, giving back a string of gains posted earlier in the week. Weakness was broad-based, spreading across the technology, consumer durables, non-energy minerals and healthcare sectors. Technology names led the losses, with Xero falling 4.5%, WiseTech Global dropping 4.6%, and Megaport sliding 3.3%. Gold miners also retreated, with Northern Star Resources down 3.9% and Evolution Mining off 2.4%. Australia’s four major banks were a rare bright spot, rising between 1% and 1.5% as investors rotated toward more defensive, income-generating stocks.

For the week overall, the index shed roughly 0.3%, marking its third consecutive weekly decline even as trading earlier in the week had briefly pushed the market toward stronger gains.

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Trump’s tariff announcement rattles sentiment

The pullback came after the Trump administration said it would impose new tariffs on 60 trading partners, a move that unsettled global markets and flowed through to Australian trading given the country’s close economic and trade ties with both the United States and Asia-Pacific export markets. The announcement contributed to a sharp overnight selloff on Wall Street, which set a negative tone for Friday’s session in Sydney. The Dow Jones Industrial Average fell 0.97% overnight, while the tech-heavy Nasdaq Composite dropped a steeper 2.15%, dragging down sentiment across Asia-Pacific markets the following morning.

Strong jobs data complicates the rate outlook

Domestically, robust employment figures added another layer of complexity to the week’s trading. Australia added 76,000 jobs in June, far exceeding consensus expectations of around 15,000, while the unemployment rate held steady at 4.4%. The stronger-than-expected labor market data initially helped push the index higher earlier in the week, with the ASX 200 climbing as much as 1.1% intraday on Thursday to touch 8,926.30, its best level since mid-June, before those gains were pared back as investors recalibrated expectations for Reserve Bank of Australia policy.

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The strong jobs report lifted the odds of an August RBA rate increase to roughly 36%, with markets now largely pricing in a move to 4.6% by the end of the year following three rate hikes already delivered in 2026. That shift added pressure to rate-sensitive sectors, including parts of the financial sector, even as the broader market weighed the implications of a still-resilient labor market against the risk of further tightening.

With Australia’s inflation data for June and the second quarter due out the following week, investors remained cautious about the potential for persistent price pressures to further complicate the central bank’s policy path heading into the back half of the year.

Commodities offer a partial offset

Mining and materials stocks provided some support during the week, helped by strength in key commodity prices. Gold traded around $4,116 an ounce, while iron ore futures climbed 1.7% to $98.70 in Singapore, lifting major miners including BHP Group, which rose 1.5% to $60.63, Fortescue, up 1% to $18.76, and Northern Star Resources, up 2% to $20.74 during Thursday’s session before the sector cooled into Friday’s close.

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Energy stocks also found support mid-week after oil prices rose 2.3% to $96.24 a barrel, following reports that Iran-backed Houthi militants had attacked two Saudi Arabian oil tankers in the Red Sea, adding a geopolitical risk premium to crude markets that flowed through to Australian energy shares.

A market still near record territory despite the pullback

Even with the week’s decline, the ASX 200 remains within striking distance of the record highs it set earlier this year. The index touched an all-time intraday high of 9,198.6 points in February before pulling back toward the high-8,000s range by mid-year. Over its more than 25-year history, the benchmark index has delivered a long-term annualized total return of roughly 8.2%, including dividends, making short-term pullbacks like the one seen this week a routine part of its longer-term trajectory rather than a departure from it.

Seasonally, July has historically been one of the stronger months for the ASX 200, with the index averaging a gain of roughly 2.13% for the month since 1980 and finishing higher in 72% of those years. Recent Julys in particular have performed well, with the index closing higher in 11 of the last 12 years during the month, making this year’s choppier trading somewhat of an outlier relative to the seasonal pattern.

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What investors are watching next

With inflation data for June and the second quarter due the following week, market participants are likely to remain focused on how that report shapes expectations for the Reserve Bank of Australia’s next policy move. The interplay between a resilient labor market, persistent inflation risk, and the fallout from the latest round of U.S. tariff actions is expected to remain the dominant theme driving Australian equity markets in the near term, alongside ongoing volatility in global commodity prices and continued swings in U.S. technology shares, which have had an outsized influence on sentiment in Sydney trading throughout the year.

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ETMarkets AIF Talk | Next-generation entrepreneurs will create India’s biggest wealth opportunities: Hiren Ved

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ETMarkets AIF Talk | Next-generation entrepreneurs will create India's biggest wealth opportunities: Hiren Ved
India’s next wave of wealth creation may not come solely from established listed companies but increasingly from a new generation of entrepreneurs building businesses in strategic sectors such as artificial intelligence (AI), semiconductors, defence, healthcare, data centres and green energy, believes Hiren Ved, Director and CIO at Alchemy Capital Management.

In an interaction with Kshitij Anand of ETMarkets, Ved said India’s evolving innovation ecosystem is creating a fertile ground for founders who are leveraging advanced technologies to build globally competitive businesses with strong growth potential and attractive economics.

He believes investors with a long-term horizon should look beyond conventional equity strategies and consider opportunities across listed and unlisted markets to capture this emerging wealth creation cycle.

Ved also shared his outlook on small-cap investing, market volatility, IPO and pre-IPO opportunities, the role of Category III AIFs in HNI portfolios, and why India’s AI and data centre ecosystem could emerge as one of the country’s most compelling long-term investment themes. Edited Excerpts –

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Q) Thanks for taking the time out. Please take us through the performance of the fund from short- & long-term perspective.

A) The investment approach has continued to demonstrate balanced performance despite a volatile market environment.

Alchemy Long Term Ventures Fund has delivered an absolute return of 17.0% in CY2026 year-to-date basis and a CAGR of 22.6% since its inception on 1 September 2023, reflecting the fund’s investment philosophy to identify the businesses with strong earnings momentum, robust balance sheets, and favourable sectoral tailwinds.

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Alchemy Long Term Ventures Fund, Series 2 has also delivered a CAGR of 14.8% since its launch on 1 September 2025, reinforcing the consistency of our investment approach across market cycles.

Data as on 30 June 2026.
(Returns are net of Post Fees, Expenses and Taxes. Consolidated Returns are calculated using unitization method. The Consolidated Returns may vary with investors’ returns depending on the class/series investor have subscribed into. |Past performance is not indicative of the future performance. Returns less than 1 Year: Absolute, greater than 1 Year: CAGR| Performance related information provided herein is not verified by SEBI.)
Q) The fund primarily focuses on small-cap opportunities while retaining the flexibility to invest up to 35% in unlisted securities. How do you balance the higher growth potential of these segments with liquidity and valuation risks?
A) Alchemy Long Term Ventures Fund, Series 3 (Category III AIF) is designed for sophisticated investors who have a long-term outlook and understand the risks associated with such a strategy.

Clearly, this is for investors that have an appetite for risk but are looking for differentiated exposure beyond traditional listed equity strategies. The unlisted sleeve is to take advantage of dynamism and entrepreneurial energy of entrepreneurs building the next generation of high growth businesses in strategic growth sectors.

We aim to blend that with similar companies operating in these sectors in the listed space. The ability to straddle both listed and private opportunities gives us significant flexibility to deploy capital. Restricting unlisted to up to 35% of the fund, allows us to strike a balance between liquidity and tenure of investments which is between 4-5 years.

We are reasonably sensitive about entry valuations. On the listed side we use market volatility to our advantage as we always have liquidity on tap.

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Q) The strategy identifies themes such as data centres & AI, defence, green mobility, semiconductors, manufacturing and green energy as key opportunities. Which of these themes do you believe could create the most wealth over the next 4-5 years, and why?
A) All the above sectors have tremendous potential, in my view. Our idea is to identify companies and founders in the value chain that exhibit both – great growth opportunity combined with attractive business economics, in terms of profitability and economic returns.

One without the other does not cut it with the objectives of the strategy. It is our firm belief that some of the most innovative high growth companies may be built by the next generation of entrepreneurs who are unconstrained by previous profit pools and are likely to embrace advanced technologies and practices faster than incumbents.

The opportunity set for these companies arises from building certain foundational capabilities in defence, aerospace, space, semiconductors, healthcare, biotech, AI, data centre infrastructure, green energy and green mobility to name a few.

Q) The existing Alchemy Long Term Ventures Fund has delivered a 22.6% post-tax CAGR since inception, with significant exposure to industrials and IT. What have been the key drivers of this performance, and can the same investment framework be replicated in Alchemy Long Term Ventures Fund, Series 3?
A) Our exposure in Alchemy Long Term Ventures Fund has been towards manufacturing and industrial companies spanning several strategic sectors. In IT, our exposure has not been to traditional services companies but in companies that leverage specialised skill sets in silicon design, space communication, devices and transportation sectors.

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We have also seen encouraging progress from our investments in healthcare companies bringing original research molecules to commercial stage. Yes, we intend to carry the same investment philosophy in Alchemy Long Term Ventures Fund, Series 3 as well.

The portfolio performance was supported by a combination of earnings growth and valuation re-rating of our portfolio companies.

Q) The strategy can invest across listed equities, IPO anchor books, pre-IPO opportunities, and unlisted securities. In the current market environment, where are you finding the most attractive risk-reward opportunities?
A) Market volatility always gives us many attractive entry points. March 2026 was one such occasion. In anchor books and unlisted we are extremely selective in our approach. We continue to see a healthy interest from IPO-bound companies to be part of their cap table, a trend we attribute to our long-standing presence and historical track record.

In unlisted, the quality of the founders and entry valuations matter the most, so we are always on a lookout for that.

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Q) Given the minimum investment of Rs 1 crore and the fund’s four-year tenure, what role should a Category III AIF such as this play in an HNI investor’s overall portfolio?
A) Alchemy Long Term Ventures Fund, Series 3 is a high-risk strategy, and investors should consider such investments in the context of their investment objectives, liquidity needs and risk tolerance. Investors may consider making investments of such sums with a horizon of over the next 5-10 years. This would generally sit between small/midcap equity and private equity.

Q) India’s data centre capacity per 1,000 internet users remains significantly below China and the US, according to the fact sheet. Where do you see the biggest investment opportunities emerging as India builds its AI and data centre infrastructure?
A) I think the initial opportunities are at an infrastructure layer and its entire ecosystem – from designing, building to equipment that go into a data centre.

However, we are now seeing many interesting opportunities even on the application layer, where companies are using AI models and capabilities to deliver services to enterprise customers and consumers. We have made select investments in companies leveraging AI at the application layer.

Disclaimer: Past performance is not indicative of the future performance. The sectors herein are solely for information purposes and may or may not form part of the Fund’s portfolio at the time of making investments.

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Alchemy Long Term Ventures Fund, Series 3 is one of the schemes of Alchemy Alternative Investment Trust, registered with Securities and Exchange Board of India (SEBI) as a Category III – Alternative Investment Fund, vide registration number IN/AIF3/17-18/0381. Alchemy Capital Management is registered with the Securities and Exchange Board of India (SEBI) as a Portfolio Manager and appointed as the Investment Manager of the Trust and the Fund.

(Disclaimer: Recommendations, suggestions, views, and opinions given by experts are their own. These do not represent the views of the Economic Times)

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Gold rises on weaker dollar; traders weigh Middle East pause, Fed outlook

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Seattle shooting leaves 2 dead, 5 injured, according to reports

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Botanix Pharmaceuticals Limited (BXPHF) Discusses Quarterly Activity, Cash Flow, and Commercial Progress of Sofdra for Primary Axillary Hyperhidrosis Transcript

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OneWater Marine Inc. (ONEW) Q1 2026 Earnings Call Transcript

Jane Morgan

Good morning, everyone, and thank you for joining today’s investor webinar for Botanix Pharmaceuticals with the ASX ticket code BOT. My name is Jane Morgan, the Investor and Media Relations Manager. And today, I am joined by our Executive Chairman, Vincent Ippolito, our CEO, Dr. Howie McKibbon; and our U.S. CFO, Chris Lesovitz.

So for those who are new to the Botanix story, Botanix is a commercial stage dermatology company operating in Australia and the United States with this FDA-approved product Sofdra, which is available in America. Sofdra is a prescription-only topical gel medicine, which used to treat excessive underarm sweating or more formally known as primary axillary hyperhidrosis. It is used in treating adults and children aged 9 and over.

The company has transitioned from a development stage business into a revenue-generating commercial entity with Sofdra as its primary growth driver. Today’s presentation will be followed by a Q&A session. [Operator Instructions]

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Vince, I’m going to hand to you.

Vincent Ippolito
MD & Executive Chairman

Thank you, Jane, and a pleasant good morning to everyone here joining us from Australia. Dr. Howie McKibbon and myself are here live in Sydney for this call. Chris Lesovitz is calling in from our headquarters in the U.S. and we’re pleased to present the Botanix Quarterly Activity Report and 4C Quarterly Cash Flow Report for the period ending 30 June 2026. And the company has come a long way since our highly successful commercial launch of Sofdra a little over a year ago, and we’re very pleased with

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Earnings call transcript: Delta Electronics Thailand posts strong Q2 2026 growth

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Akzo Nobel: Axalta Merger Beats Nippon's Bid

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Akzo Nobel: Axalta Merger Beats Nippon's Bid

Akzo Nobel: Axalta Merger Beats Nippon's Bid

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Negative Breakout: These 6 stocks cross below their 200 DMAs

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The Economic Times

In the NSE list of stocks with a market cap over Rs 10,000 crore, the closing prices of six stocks crossed below their 200 DMA (Daily Moving Averages) on July 24, according to stockedge.com’s technical scan data. Trading below the 200 DMA is considered a negative signal because it indicates that the stock’s price is below its long-term trend line. The 200 DMA is used as a key indicator by traders for determining the overall trend in a particular stock. Take a look:​

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Botanix Pharmaceuticals Limited (BXPHF) Discusses Quarterly Activity, Cash Flow, and Commercial Progress of Sofdra for Primary Axillary Hyperhidrosis – Slideshow (OTCMKTS:BXPHF) 2026-07-26

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OneWater Marine Inc. (ONEW) Q1 2026 Earnings Call Transcript

This article was written by

Seeking Alpha’s transcripts team is responsible for the development of all of our transcript-related projects. We currently publish thousands of quarterly earnings calls per quarter on our site and are continuing to grow and expand our coverage. The purpose of this profile is to allow us to share with our readers new transcript-related developments. Thanks, SA Transcripts Team

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