Atrium Therapeutics Launches with Approximately $270 Million to Advance Novel RNA Medicines for Rare Genetic Cardiomyopathies [Accessibility Statement] Skip NavigationSpinoff from Novartis AG's acquisition of Avidity Biosciences advances precision cardiology programs using targeted RNA delivery platformLead candidates ATR 1072 and ATR 1086 expected to enter clinical trials for PRKAG2 syndrome and PLN cardiomyopathy, respectivelySAN DIEGO, Feb. 27, 2026 /PRNewswire/ -- Atrium Therapeutics, Inc. (Nasdaq: RNA) ...

SAN DIEGO, Feb. 26, 2026 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) today announced highlights from Day 3 of Bionano Symposium 2026, entitled OGM Making its Mark in Constitutional Genetic Disorders. Presentations highlighted how these conditions, which include developmental delay, intellectual disability, neuromuscular disorders, reproductive disorders, birth defects and other so-called rare diseases, present unique challenges to the cytogenetics and molecular pathology teams investigating th ...

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for BEAM‑304 Anticipated in 2026 Updated Phase 1/2 Data and Next Steps for Pivotal Development for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) on Track for Q1 2026 Strategic Financing Agreement with Sixth Street Provides up to $500 Million in Long-term, Non-dilutive Capital to Fund Anticipated Launch of Risto-cel in Sickle Cell Disease (SCD); U.S. Biologics License Appl ...

CAMBRIDGE, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will announce its fourth quarter and full year 2025 financial results and a new liver-targeted genetic disease program on an investor webcast on Tuesday, February 24, 2026, at 8:00 a.m. ET.The live webcast will be available in the investor section of the company's website at www.beamtx.com and will be ...

Core Insights - Beam Therapeutics has reached alignment with the U.S. FDA on a potential accelerated approval pathway for BEAM-302, a treatment for Alpha-1 Antitrypsin Deficiency (AATD), based on biomarker endpoints [1][4] - The company expects to submit a Biologics Licensing Application (BLA) for risto-cel (formerly BEAM-101) as early as the end of 2026 [1][13] - Beam's cash position is estimated at $1.25 billion as of December 31, 2025, providing an operating runway into 2029 to support the anticipated launch of risto-cel and the execution of the BEAM-302 pivotal development plan [10][11] Liver-targeted Genetic Disease Franchise - Beam is developing a platform for precision gene editing therapies targeting liver diseases, utilizing lipid-nanoparticle delivery systems [3] - BEAM-302 aims to be a first-in-class therapy for AATD, demonstrating clinical proof of concept in an ongoing Phase 1/2 trial with over 25 patients treated [4] - The company plans to enroll approximately 50 additional patients for the expansion of the Phase 1/2 study to support the BLA submission [4] Hematology Franchise - Risto-cel is an investigational autologous cell therapy for sickle cell disease (SCD), showing a differentiated treatment profile in the ongoing BEACON Phase 1/2 clinical trial [8] - The therapy has demonstrated a deeper resolution of SCD markers and reduced hospital time, with a robust manufacturing process [8] - Beam is also focusing on next-generation programs for SCD, including in vivo delivery strategies and the development of the ESCAPE platform for non-genotoxic treatments [9] Financial Position and Future Outlook - The estimated cash and marketable securities position of $1.25 billion will support Beam's operating expenses and capital requirements through 2029 [10][11] - The company is well-positioned to advance its pipeline of genetic therapies, with a focus on regulatory execution and clinical data generation [2][11] - Beam plans to announce a new program for its liver-targeted genetic disease franchise in the first half of 2026 [6]

Core Insights - Seer, Inc. announced a significant genome-wide association study (GWAS) published in Nature Genetics, demonstrating the importance of mass spectrometry validation in establishing reliable drug targets and clinical biomarkers [1][10] Group 1: Study Overview - The study involved approximately 1,600 blood samples from diverse ethnic backgrounds, with a discovery cohort of 1,260 and a replication cohort of 325, utilizing Seer's Proteograph workflow [2] - A total of 5,753 proteins were detected, with 1,980 quantified in at least 80% of participants [2] Group 2: Findings and Implications - Researchers identified 364 protein quantitative trait loci (pQTLs) associated with protein abundance, with 102 replicating in the independent cohort, including 35 previously unreported signals [3] - The study highlighted that up to one-third of protein-gene associations reported by affinity-based assays do not replicate, underscoring the necessity for accuracy in proteogenomics [4][7] Group 3: Methodology and Technology - Seer's Proteograph platform employs mass spectrometry to measure proteins directly at the peptide level, mitigating confounding effects from affinity reagents [5][6] - The study compared mass spectrometry results with two major affinity-based proteomics resources, confirming that mass spectrometry is crucial for reliable proteomic analysis [6] Group 4: Future Directions - The findings strengthen confidence in drug discovery and biomarker development by ensuring that selected targets reflect genuine biological signals rather than technical noise [9] - The study positions proteomics as a population-ready science, essential for supporting drug targets and biomarkers with the rigor needed for clinical impact [11]

Core Insights - Clairity, Myriad Genetics, and MagView have formed a collaboration to enhance breast cancer risk assessment for women, aiming to fill a significant gap in identifying high-risk individuals without adding administrative burdens [1][2][7] Company Summaries Clairity - Clairity is an AI-powered precision health company that has developed Clairity Breast, the first FDA-authorized tool to estimate a woman's five-year breast cancer risk from a routine mammogram, focusing on prevention rather than detection [3][6] - The company aims to empower health systems and radiologists to act earlier in cancer care, potentially saving lives and reducing overall healthcare costs [3] Myriad Genetics - Myriad Genetics specializes in molecular diagnostic testing and precision medicine, offering tests that assess disease risk and guide treatment decisions, thereby improving patient care and reducing healthcare costs [4][6] - The integration of Myriad's MyRisk Hereditary Cancer Test with Clairity's AI-powered assessment is expected to provide clinicians with actionable insights for earlier and more personalized interventions against breast cancer [2][7] MagView - MagView is a leader in breast imaging workflow solutions, providing software that enhances mammography reporting and high-risk screening, utilized by over 2,500 facilities across the U.S. [5][6] - The Luminary Risk platform from MagView will integrate Clairity and Myriad's offerings, streamlining risk information capture and improving workflow efficiency for breast health management [2][5]

Core Insights - Palvella Therapeutics has announced QTORIN™ pitavastatin as a new product candidate for the treatment of disseminated superficial actinic porokeratosis (DSAP), a serious skin disease with no FDA-approved therapies available [1][3] - The company plans to initiate a Phase 2 clinical trial for QTORIN™ pitavastatin in the second half of 2026, following discussions with the FDA [1][3] Company Overview - Palvella Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for serious, rare skin diseases without FDA-approved treatments [5][6] - The company utilizes its patented QTORIN™ platform to develop novel topical product candidates, with QTORIN™ pitavastatin being the second product candidate in its pipeline [5][6] Disease Context - DSAP is characterized by persistent lesions that can lead to chronic skin integrity loss and has the potential to transform into squamous cell carcinoma [2] - There are over 50,000 diagnosed patients in the U.S. suffering from DSAP, highlighting a significant unmet medical need [2]

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]

Core Insights - Microbix Biosystems Inc. and EMQN CIC have launched a program to enhance the identification of patients with gene variants affecting the metabolism of Clopidogrel, a widely prescribed antiplatelet drug [1][2] Group 1: EQA Program Details - The new External Quality Assessment (EQA) program aims to ensure proficiency in Point-of-Care Tests (POCTs) that identify three gene variants of the Cytochrome P450 CYP2C19 enzyme, which are critical for determining appropriate Clopidogrel dosing [2][4] - Over 20% of individuals carry these gene variants, which can lead to either overdosing or underdosing of Clopidogrel, posing significant health risks [2][3] Group 2: Market Context - Clopidogrel is prescribed to over 15 million patients annually in the U.S. alone, indicating a substantial market for pharmacogenomic testing to optimize treatment and minimize risks associated with improper dosing [3] - The EQA program will provide participants with three samples every two months, totaling 18 samples per year, to ensure ongoing accuracy in genetic testing [4] Group 3: Company Contributions - Microbix is supplying quality assessment products (QAPs™) for the EQA program, which will assist in evaluating the accuracy of genetic tests and support safe prescription practices [4][5] - The collaboration with EMQN and Copan aims to enhance the quality of genetic testing, with Microbix expanding its role in the field of pharmacogenomics alongside its established presence in infectious diseases and oncology [5][7] Group 4: Company Background - Microbix Biosystems Inc. is a life sciences innovator with over 120 employees, generating revenues of approximately C$2.0 million or more per month, and is involved in manufacturing critical ingredients for the global diagnostics industry [7][8] - The company is ISO 9001 & 13485 accredited and has a presence in over 30 countries, supported by a network of international distributors [8]