Genetic Technologies(GENE)
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Century Therapeutics to Present at Chardan's 9th Annual Genetic Medicines Conference
Globenewswire· 2025-10-14 20:01
Core Insights - Century Therapeutics, Inc. is focused on developing induced pluripotent stem cell (iPSC)-derived cell therapies targeting autoimmune diseases and cancer [1][3] - Chad Cowan, Ph.D., the Chief Scientific Officer, will present at Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025 [1] - The company utilizes its proprietary Allo-Evasion™ technology to enhance immune evasion in its therapies, aiming to improve patient access and treatment outcomes [3] Company Overview - Century Therapeutics is a clinical-stage biotechnology firm listed on NASDAQ under the ticker IPSC [3] - The company is advancing a pipeline of iPSC-derived cell therapies, which are designed to address significant medical needs in autoimmune diseases and cancer [3] - The therapies are produced from the company's iPSC cell foundry, indicating a focus on innovative and scalable treatment solutions [3] Presentation Details - The presentation by Chad Cowan will take place at 12:20 p.m. ET in New York, NY [1] - A live webcast of the presentation will be accessible on the company's Investors page, with an archived replay available for at least 30 days [2]
Microbix Assisting EMQN with Second Genetic-Test EQA Program
Globenewswire· 2025-09-23 11:00
MISSISSAUGA, Ontario and MANCHESTER, United Kingdom, Sept. 23, 2025 (GLOBE NEWSWIRE) -- Microbix Biosystems Inc. (TSX: MBX, OTCQX: MBXBF, Microbix®), a life sciences innovator, manufacturer, and exporter, and EMQN CIC (EMQN), a global supplier of laboratory external quality assessment (“EQA”) schemes for human genomic testing, announce the go-live of a program to support rapid and accurate identification of patients who carry gene-variants associated with faster and slower than normal metabolism of the wide ...
Regeneron Announces Positive Phase 3 Trial in Adults with Ultra-Rare Genetic Disorder Fibrodysplasia Ossificans Progressiva (FOP), Demonstrating that Garetosmab Prevents Greater than 99% of Abnormal Bone Formation
Globenewswire· 2025-09-17 11:00
Core Insights - Garetosmab is the first and only treatment to show significant reduction in both the number and volume of abnormal bone lesions in adults with fibrodysplasia ossificans progressiva (FOP) [1][2][6] - The Phase 3 OPTIMA trial met its primary endpoint, demonstrating a 90% or greater reduction in new heterotopic ossification (HO) lesions at 56 weeks [1][3][4] - The Independent Data Monitoring Committee recommended transitioning placebo patients to garetosmab, with U.S. regulatory submission planned for year-end 2025 [1][6] Trial Details - The OPTIMA trial was a global, multi-center, randomized, double-blind, placebo-controlled study involving 63 adults with FOP [2][9] - Participants received either placebo, 3 mg/kg garetosmab, or 10 mg/kg garetosmab every four weeks for 56 weeks [2][9] - Key secondary endpoints included a significant reduction in clinician-assessed flare-ups, with an 89% reduction in the 10 mg/kg group compared to placebo [4][5] Efficacy Results - At 56 weeks, the total number of new HO lesions was reduced by 94% in the 3 mg/kg group and 90% in the 10 mg/kg group compared to placebo [4][5] - The mean total volume of new HO lesions showed a reduction of 99% in both garetosmab treatment arms compared to placebo [4][5] Safety Profile - There were no discontinuations in the garetosmab treatment arms, with one discontinuation in the placebo arm due to an ovarian cyst [5] - A dose-dependent increase in skin and soft tissue infections was observed, but no serious bleeding events were reported [5] - Serious treatment-emergent adverse events occurred in a small number of patients across all treatment groups, with no deaths reported [5] Future Plans - A Phase 3 trial of garetosmab in adolescents and children with FOP, named OPTIMA 2, is planned to begin next year [6][11] - Global regulatory submissions for garetosmab are slated for 2026 following the U.S. submission [6]
干货:起底蛋白转印“绊脚石”,突破实验瓶颈
仪器信息网· 2025-09-02 03:58
Core Viewpoint - The article emphasizes the importance of efficient and reliable protein transfer techniques in Western Blotting, highlighting the challenges faced in traditional methods and introducing innovative solutions to enhance laboratory efficiency [1][24]. Summary by Sections Protein Transfer Challenges - Common issues in protein transfer include faint bands due to improper transfer time, high background noise from inadequate membrane handling, and inconsistent results caused by aging transfer equipment [3][4]. - These problems can lead to increased experimental repetition, wasted samples and reagents, and potential misinterpretation of results, ultimately affecting research progress [4]. Protein Transfer Methods - The article outlines three main types of protein transfer methods: wet transfer, semi-dry transfer, and dry transfer, each with its own advantages and limitations [5][6][10][14]. Wet Transfer - Wet transfer is a traditional method that requires a specific assembly order and is time-consuming, typically taking at least one hour [6][7]. - It is suitable for a wide range of protein sizes and complex samples but involves the use of toxic reagents, increasing laboratory waste [7]. Semi-Dry Transfer - Semi-dry transfer reduces transfer time to about 10 minutes but has limitations in transfer efficiency for large and small proteins [10][11]. - It requires careful control of experimental conditions and may necessitate additional optimization for different samples [11]. Dry Transfer - Dry transfer utilizes specialized transfer membranes and is faster but incurs higher long-term costs due to the price of consumables [14][15]. - It is sensitive to protein characteristics, making it less effective for extreme molecular weights [15]. Considerations for Protein Transfer - The choice of transfer method should be based on the specific requirements of the experiment, such as protein size and sample complexity [18]. - Proper preparation of the gel and membrane, as well as the use of fresh transfer buffer, are critical for successful protein transfer [19][21]. Innovations in Protein Transfer - The introduction of the eBlot™ rapid wet transfer system by Kingsray addresses traditional wet transfer issues, combining stability and speed to achieve efficient protein transfer in 5-15 minutes [24][25]. - The system is designed to enhance laboratory efficiency while maintaining high transfer quality, making it a valuable tool for researchers [24]. Promotional Activities - Kingsray is currently running a "trade-in" program for upgrading to the eBlot™ system, offering discounts based on the condition of old equipment and providing additional support for users [26][28].
Ionis Posts Surprise Q2 Profit, Double Revenues On Stronger Sales From Newly Approved Genetic Disease Drug
Benzinga· 2025-07-30 18:34
Core Insights - Ionis Pharmaceuticals reported strong second-quarter results with adjusted earnings of 85 cents, a significant improvement from an adjusted loss of $35 million a year ago, and beating analysts' expectations of a 52-cent loss [1] - Revenue for the first quarter of 2025 increased by 10% to $132 million, surpassing the consensus estimate of $125.32 million, driven by higher commercial revenue [1] Revenue Performance - Ionis' revenues more than doubled to $452 million, exceeding the consensus of $282.95 million, primarily due to the successful launch of Tryngolza and increased royalty and R&D revenues [2] - Tryngolza generated net product sales of $19 million in the second quarter of 2025 and $26 million in the first half of 2025 [2] Product Developments - Tryngolza received a positive opinion from the Committee for Medicinal Products for Human Use, with a European Commission decision expected by Q4 2025 [3] - Wainua generated sales of $44 million in the second quarter of 2025, resulting in royalty revenue of $10 million [3] - Spinraza generated global sales of $393 million, leading to royalty revenue of $54 million [4] Financial Guidance - Ionis raised its fiscal 2025 sales guidance from $725 million-$750 million to $825 million-$850 million, compared to the consensus estimate of $754.67 million [6] - The company expects a 2025 adjusted operating loss of $300 million-$325 million, an improvement from previous guidance of less than $375 million [7] Market Reaction - Following the announcement, Ionis stock rose by 5.23% to $43.65 [7]
Rhythm Pharmaceuticals: Rare Genetic Obesity Pioneer With Excellent Prospects
Seeking Alpha· 2025-06-30 15:00
Group 1 - Rhythm Pharmaceuticals, Inc. (NASDAQ: RYTM) has experienced a significant increase of approximately 28% since being rated as a "Buy" in February 2025 [1] - The focus of the analysis is on the company's position in the genetic obesity market, highlighting its potential for growth and investment opportunities [1] - The author emphasizes the importance of learning from both investment successes and failures, indicating a long-term commitment to research and analysis in the healthcare sector [1] Group 2 - The article reflects a personal investment in Rhythm Pharmaceuticals, indicating a beneficial long position in the shares [2] - The author expresses a commitment to sharing insights and experiences related to investing, particularly in healthcare stocks, which may provide valuable context for potential investors [1][2] - The analysis is based on years of focused research, suggesting a depth of knowledge regarding the company's operations and market dynamics [1]
What Regeneron's Purchase Of 23andMe Means For Your Genetic Privacy
Forbes· 2025-05-21 20:35
Core Viewpoint - Regeneron Pharmaceuticals is acquiring bankrupt genomics firm 23andMe for $256 million, aiming to utilize its extensive DNA database while committing to ethical standards in data privacy [1][2][4]. Company Overview - 23andMe, once valued at $6 billion, filed for bankruptcy in March 2025 due to declining consumer interest and a significant data breach that affected millions [3]. - The acquisition is seen as a strategic move for Regeneron to enhance its genomic-driven drug discovery processes using 23andMe's database of over 15 million DNA samples [2][3]. Data Privacy Commitments - Regeneron has pledged to adhere to 23andMe's existing privacy guidelines and relevant data protection regulations, ensuring that personal data will not be used for research without explicit consent [4][5]. - An independent overseer will evaluate the deal's impact on consumer privacy, with a report due to the bankruptcy court in June [4]. Regulatory and Ethical Concerns - The acquisition has raised concerns among privacy advocates regarding the handling of genetic information, especially in light of past data breaches [4][10]. - U.S. lawmakers are monitoring the situation, worried that genetic data could be sold to entities with different ethical standards [10]. Future Implications - The deal highlights broader questions about ownership and usage of personal genetic data, emphasizing the potential risks associated with sharing such information [13][14]. - As the acquisition moves toward finalization in the third quarter of 2025, the fundamental question remains whether the benefits of contributing to medical research outweigh the privacy risks [14][15].
Abeona Surges After FDA Clears First Gene Therapy For Severe Genetic Skin Disease
Benzinga· 2025-04-30 20:41
Core Insights - Abeona Therapeutics Inc. received FDA approval for Zevaskyn, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][3] - Zevaskyn is a single-application treatment that utilizes genetically modified skin cells to produce functional Type VII collagen, allowing for significant coverage of wounds [2][3] - The list price for Zevaskyn is set at $3.1 million per treatment, significantly higher than previous estimates, with projected sales of over $30 million by the end of 2025 [5][6] Market Potential - HC Wainwright estimates peak U.S. sales for Zevaskyn could reach around $600 million, with a potential market opportunity exceeding $2 billion if all 750 identified patients are treated [6] - The updated revenue forecast for Abeona is $31.6 million in 2025, increasing to $130.4 million in 2026 [5][6] Competitive Landscape - Zevaskyn may be used in conjunction with other treatments, such as Krystal Biotech's Vyjuvek, which was recently approved in Europe for similar indications [3][4] - Analyst Raghuram Selvaraju maintains a Buy rating for Abeona, raising the price target from $15 to $20 per share, reflecting increased confidence in Zevaskyn's market position [6]
Genetic Screening Testing for the Preventive Health Market, 2025-2035 Analysis - Dominated by Exact Science, Labcorp Genetics, Blueprint Genetics, Ambry Genetics, and Genex Diagnostics
GlobeNewswire News Room· 2025-04-11 09:32
Dublin, April 11, 2025 (GLOBE NEWSWIRE) -- The "Genetic Screening Testing for Preventive Health Market - A Global and Regional Analysis: Focus on Test Type, Technology, Application, End User and Country-Level Analysis - Analysis and Forecast, 2025-2035" report has been added to ResearchAndMarkets.com's offering.The market for genetic screening testing in preventive health has been experiencing rapid growth, driven by driving factors such as technological advancements, increased consumer interest in persona ...
23andMe's Bankruptcy Sparks Privacy Concerns Over Genetic Database Of 15 Million Customers
Benzinga· 2025-03-25 18:45
Core Points - 23andMe Holding Co has voluntarily filed for Chapter 11 bankruptcy to facilitate a sale process aimed at maximizing business value following a rejected acquisition offer from CEO Anne Wojcicki and her affiliates [1] - The company has proposed an auction for its assets on May 14, raising concerns about the potential sale of customer genetic data due to bankruptcy [2] - 23andMe maintains that the Chapter 11 filing does not alter how it stores, manages, or protects customer data, and customers will retain access to their accounts and genetic reports during the process [3] Data Privacy Concerns - Customers' genetic data may be sold to the highest bidder, prompting concerns regarding privacy and legal protections [2] - Bidders in the auction must comply with the company's privacy practices, which do not restrict the transfer of personally identifiable information during bankruptcy or asset sales [4] - New York Attorney General issued a consumer alert, providing instructions for customers on how to delete their personal data and destroy genetic samples held by the company [3] Recent Incidents - In 2023, a data breach occurred when a threat actor accessed several accounts through credential stuffing, with the company learning of the breach in October 2023 [5] - The company settled lawsuits related to the breach for $30 million, but objections have been raised regarding the settlement's design to undermine claimants' rights [6] - Following the bankruptcy news, 23andMe's stock price fell by 11.7%, trading at $0.64 [6]