Jacob Funds, a mutual fund company, released its fourth-quarter 2025 investor letter. A copy of the same can be downloaded here. After an optimistic quarter, equity markets corrected slightly in November. Although overall economic growth has been robust throughout the year, recent divergent data on consumer spending and the labor market have put doubt on future projections. This pattern has become somewhat familiar over the past few years, as conflicting economic data has become the norm. As for the Fund’s ...

Core Viewpoint - The gene editing sector experienced a significant setback following a severe liver injury incident in a patient treated with Intellia Therapeutics' NEX-z therapy, leading to a 42.23% drop in the company's stock price and a nearly 50% loss in market capitalization [1][2]. Group 1: Incident Details - A patient with a rare heart disease undergoing NEX-z treatment reported severe liver damage, prompting the suspension of two Phase III clinical trials [1][3]. - This incident marks the second occurrence of Grade 4 liver toxicity related to NEX-z this year, raising concerns about the safety of gene editing therapies [1][4]. - The first incident in May was asymptomatic and self-resolving, contrasting sharply with the current case, which required hospitalization [1][3]. Group 2: Market Impact - The safety concerns surrounding NEX-z have negatively affected the entire gene editing sector, causing stock declines in other companies such as Beam Therapeutics and Prime Medicine [1][4]. - Analysts had previously viewed the risk/reward balance of NEX-z favorably, but the recent events have led to increased scrutiny and skepticism regarding its safety profile [3][4]. Group 3: Market Potential and Competition - The market for ATTR-CM, the condition treated by NEX-z, is projected to exceed $11.2 billion by 2030, indicating significant commercial potential [2]. - Other treatments for ATTR-CM, such as Alnylam's Amvuttra, have shown strong sales growth, highlighting the competitive landscape and the urgency for Intellia to address safety concerns [8][9]. Group 4: Future Considerations - Intellia is collaborating with liver disease experts and regulatory bodies to investigate the recent adverse event and explore potential risk management strategies [6][7]. - The company faces financial pressure, with cash flow projected to last until 2027, making the resolution of these safety issues critical for its future [7][8].

Summary of the Genetic Medicines Conference - Editas Medicine Industry Overview - The conference focused on the genome editing industry, highlighting advancements in technologies and programs related to gene editing therapies [1][2] - The discussion included various companies involved in the field, such as Editas Medicine, Arbor Bio, Prime Medicine, Yultec Therapeutics, and GenEdit Bio [2] Key Points and Arguments Company Introductions - **Yultec Therapeutics**: Focuses on in vivo genome editing therapies with five clinical programs, targeting diseases from ultra-rare to chronic cardiovascular conditions [4] - **Editas Medicine**: An in vivo CRISPR gene editing company with a lead program, EDIT401, showing a 90% reduction in LDL cholesterol levels, aiming for clinical trials by the end of 2026 [6] - **GenEdit Bio**: Developing affordable DNA surgeries for genetic diseases, with a focus on eye diseases and CAR T cell therapies [8][10] - **Prime Medicine**: Specializes in prime editing technology, targeting liver diseases and working on various genetic disorders [11][12] - **Arbor Bio**: Focuses on in vivo genome editing with a broad platform of editing approaches, including programs targeting the CNS [13][14] Technological Advancements - The genome editing field has expanded to include various technologies such as nuclease editing, base editing, and prime editing, with discussions on how these technologies will coexist and evolve [15][16] - The importance of product differentiation and the need for effective delivery systems were emphasized, with a consensus that delivery is as critical as the editing technology itself [28][29] Lessons Learned from Initial Therapies - The technical success rates from preclinical to clinical stages have been unprecedented, highlighting the rapid advancement of gene editing technologies [25][26] - The need for companies to focus on differentiation and the commercial viability of one-time therapies was discussed, with an emphasis on the economic arguments for such treatments [27][46] Commercial Viability and Market Considerations - The commercial case for one-time gene editing therapies was debated, with examples of partnerships and the importance of understanding the rare disease market [50][51] - The potential for gene editing therapies to transform patient care was highlighted, with a focus on the need for effective pricing strategies and understanding the current standard of care [55][61] - The discussion included the importance of low-cost delivery systems to enhance commercial viability and patient access to therapies [59][61] Future Outlook - The panelists expressed optimism about the future of gene editing, predicting multiple approvals and launches in the coming years, alongside an evolving payer landscape that may better accommodate these therapies [68] Additional Important Insights - The variability of clinical responses in edited patients is narrower compared to other therapeutic modalities, which may lead to higher responder rates and lower treatment costs [53][54] - The need for companies to adapt their business models and focus on specific commercial opportunities was emphasized, indicating a more disciplined approach in the industry [65][66] This summary encapsulates the key discussions and insights from the conference, providing a comprehensive overview of the current state and future potential of the genome editing industry.

Core Viewpoint - Prime Drink Group Corp. is initiating a rights offering to raise a minimum of $3 million, aimed at recapitalizing the company and facilitating the acquisition of Triani Canada Inc. from receivership [1][2][4]. Group 1: Rights Offering Details - The company will offer a maximum of 353,409,888 rights to existing shareholders, with each right allowing the purchase of one share at a price of $0.0825 [1][3]. - The rights offering is expected to result in a total of 706,819,776 shares outstanding if all rights are exercised, with the new shares representing 50% of the total [3]. - The rights will expire on July 25, 2025, at 4:00 p.m. Eastern time, after which unexercised rights will be void [5]. Group 2: Use of Proceeds - Proceeds from the rights offering are intended for re-acquiring Triani and for general working capital, corporate purposes, and administrative expenses [10]. - The company aims to participate in the process of re-acquiring Triani, which is currently in receivership due to creditor actions [4]. Group 3: Shareholder Participation - The rights will only be offered to shareholders residing in Canada, and registered shareholders must submit their subscription forms to the rights agent by the expiry time [6][7]. - Directors and senior officers of the company are expected to exercise a portion of their rights, although the exact number cannot be determined at this time [10].

Core Viewpoint - The acquisition of Verve Therapeutics by Eli Lilly for up to $1.3 billion has positively impacted the biotechnology sector focused on gene therapy, which has faced significant challenges recently [1] Group 1: Acquisition Details - Eli Lilly agreed to acquire Verve Therapeutics for a total price not exceeding $1.3 billion, providing a boost to the struggling gene therapy industry [1] - The acquisition led to a 74% increase in Verve Therapeutics' stock price, reflecting positive market sentiment [1] - The deal includes a contingent value right (CVR) that exceeds Verve's 30-day average stock price by over 100% [1] Group 2: Market Reactions - Companies such as Editas Medicine, Prime Medicine, Intellia Therapeutics, CRISPR Therapeutics, and Beam Therapeutics have shown improved performance following the acquisition news, despite experiencing significant declines over the past 12 months [1] - Wall Street reacted positively to Eli Lilly's acquisition proposal, indicating a favorable outlook for the gene editing sector [1] Group 3: Analyst Opinions - BMO Capital Markets expressed skepticism regarding the market demand for additional gene therapies, suggesting that Eli Lilly might have better uses for its capital [2] - Conversely, William Blair viewed the transaction as beneficial for Verve Therapeutics' shareholders, who will receive payments related to the CVR in the coming years [2] - Analyst Myles Minter stated that the acquisition is reasonable for Verve Therapeutics' shareholders and aligns well with Eli Lilly's product line [2]

Core Viewpoint - Prime Medicine has achieved a significant breakthrough in gene editing by using prime editing technology for the first time in human patients, marking a milestone in the field of biotechnology [1] Group 1: Company Developments - Prime Medicine announced the application of its prime editing therapy PM359, which aims to correct gene mutations causing chronic granulomatous disease [1] - The therapy has received initial clinical data supporting its safety and efficacy, indicating a promising advancement in treatment options for genetic disorders [1] Group 2: Industry Impact - The use of prime editing technology represents a major advancement in gene editing, potentially transforming treatment approaches for various genetic diseases [1] - Chronic granulomatous disease, which affects the functionality of immune cells including neutrophils, highlights the critical need for innovative therapies in the biotechnology sector [1]

Market Performance - The NASDAQ Biotechnology Index increased by 1.55%, closing at 4,177.9 points [1][2] - Notable gainers included Akero Therapeutics, which rose by 22.71%, and Sana Biotechnology, which increased by 22.02% [1][2] - Other significant performers were 89bio with a 20.97% rise, CureVac NV ADR up by 10.47%, and Moderna gaining 6.31% [1][2] Decliners - Anavex Life Sciences experienced a decline of 9.95%, while Prime Medicine fell by 15.01% [1][2] - Alumis saw a significant drop of 16.89% [1][2] Trading Volume - Akero Therapeutics had a trading volume of 6.77 million shares, while Sana Biotechnology traded 10.2 million shares [2] - 89bio recorded a trading volume of 7.16 million shares [2]

Core Viewpoint - Prime Editing technology, a prominent member of the CRISPR gene editing family, has made its debut in the medical field with the treatment of Chronic Granulomatous Disease (CGD) using the PM359 therapy, showing promising early clinical results [2][5][8]. Group 1: Prime Editing Technology - Prime Editing technology, developed by Professor David Liu, allows for precise gene editing without relying on DNA templates, enabling the correction of 89% of known pathogenic human genetic variations [5][7]. - The technology has been upgraded to improve editing efficiency, and it is particularly suitable for diseases like CGD, where common mutations can be corrected by inserting two missing bases in the DNA sequence [9][10]. Group 2: Clinical Trials and Results - Initial clinical data from Prime Medicine indicates that after one month of treatment with PM359, a teenage patient showed significant recovery in NADPH oxidase activity, with 66% of neutrophils fully restored, exceeding the expected clinical benefit threshold of 20% [2][8]. - The company has reported a 92% editing efficiency in correcting the most common mutation type associated with CGD in preclinical studies [7]. Group 3: Market and Economic Considerations - Prime Medicine announced a strategic restructuring, exploring external partnerships for the clinical development of PM359, highlighting the economic challenges in developing gene editing therapies for rare diseases [8][11]. - The only currently marketed gene editing therapy, Casgevy, has faced slow commercialization despite its approval, with projected sales of less than $10 million in 2024 [8]. Group 4: Future Directions - Prime Medicine plans to focus on developing gene editing therapies for hereditary liver diseases and continue supporting in vivo gene editing for cystic fibrosis, while also collaborating with Bristol-Myers Squibb on CAR-T cell therapies [11][13]. - The company aims to enhance its financial resources and accelerate innovation to ensure the widespread application of Prime Editing technology [11].

Group 1 - Twist Bioscience reported a quarterly loss of $0.66 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.56, but an improvement from a loss of $0.79 per share a year ago, indicating an earnings surprise of -17.86% [1] - The company posted revenues of $92.79 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 0.95% and showing an increase from year-ago revenues of $75.3 million [2] - Over the last four quarters, Twist Bioscience has exceeded consensus revenue estimates four times, indicating a positive trend in revenue performance [2] Group 2 - The stock has underperformed the market, losing about 15.6% since the beginning of the year compared to the S&P 500's decline of -3.3% [3] - The earnings outlook for Twist Bioscience is currently unfavorable, leading to a Zacks Rank 4 (Sell) for the stock, suggesting expected underperformance in the near future [6] - The current consensus EPS estimate for the upcoming quarter is -$0.56 on revenues of $94.77 million, and for the current fiscal year, it is -$2.14 on revenues of $374.42 million [7] Group 3 - The Medical - Biomedical and Genetics industry, to which Twist Bioscience belongs, is currently in the top 34% of Zacks industries, indicating a favorable industry outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact investor decisions [5]

SIGA Tech则收涨11.07%，CorMedix涨11.70%，Pacira Pharmaceuticals涨14.71%。 | D 名称 ÷ | | 最新 ⇒ | 醫學 → | 磨促 ⇒ | 涨跌幅 ⇒ | 交易量 | 看涨 P | 时间 ÷ | | --- | --- | --- | --- | --- | --- | --- | --- | --- | | ■ | 트 Pacira Pharmaceuticals | 26.28 | 27.64 | 25.51 | +14.71% | 3.7M | ■日 | 03:59:59 0 | | D | | | | | | | | | | | = CorMedix | 6.780 | 7.700 | 6.695 | +11.70% | 3.95M | 印刷台 | 03:59:59 0 | | D | SIGA Tech | 5.920 | 6.400 | 5.590 | +11.07% | 1.15M | ■■合 | 03:28:20 G | | L | 三 阿玛琳 | 0.430 | 0.460 | 0.396 | +5.34% | 3.05M | 图图 ...