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May 02, 2026, 15:30 ET BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society's 2026 Annual Meeting
This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is
More news about: BioMarin Pharmaceutical Inc.
May 02, 2026, 14:20 ET Palm Springs Air Museum Nominated for 4th Emmy by Pacific Southwest Chapter of the National Academy of Television Arts & Sciences
veterans, and preserving our aviation history.Open daily from 10 until 5, (closed Thanksgiving, Christmas and February 7, 2026) it is located at 745 N. Gene Autry Trail, Palm Springs, CA 92262, the main phone number is 760-778-6262. Visit PSAirMuseum.org for more info. Kids 12 and under get free admission
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May 01, 2026, 16:00 ET Foundation Fighting Blindness Names Dr. Eric Pierce as the Recipient of Its Highest Honor, the Llura Liggett Gund Award
match patients to treatments. He was among the scientists in the early clinical trials of AAV-RPE65 gene therapy — the research that led to the 2017 FDA approval of LUXTURNA®, the first in vivo gene therapy approved for an inherited disease.As principal scientist of the BRILLIANCE trial,
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May 01, 2026, 13:02 ET I AM ALS Ignites Nationwide Shift in ALS Visibility Launching Landmark Film, Celebrity PSA, and Elevating Thousands of Community Stories for Awareness Month
volunteers in front of the installation of 6,000 blue flags as we honor and remember those living with ALS, those we have lost to ALS, and those who are ALS gene carriers. WHEN:
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May 01, 2026, 09:21 ET National Pediatric Cancer Foundation Awards $1 Million Grant to Advance Breakthrough Research for Rare Pediatric Cancer
cells that work together to grow and spread. At the outer edge of these groups are "leader cells" with lower expression of the abnormal gene PAX3-FOXO1. These leader cells are the first to spread into the surrounding tissue, creating space for the mass to grow. Meanwhile, "follower
More news about: National Pediatric Cancer Foundation
May 01, 2026, 06:45 ET Ecovyst Announces Agreement to Acquire Calabrian Sulfur Dioxide & Sulfur Derivatives Business
result of new information, future developments or otherwise, except as may be required by applicable law.For more information:Gene Shiels – Director of Investor Relations(484) 617 [email protected]
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May 01, 2026, 02:30 ET Restore Vision Announces First-in-Human Clinical Interim Results for RV-001, a GPCR-Based Optogenetic Gene Therapy
ARVO and the Innovation Summit."About Restore VisionRestore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company
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Apr 30, 2026, 12:38 ET Northwest Biotherapeutics Appoints Dr. Annalisa Jenkins As Strategic Adviser To Advance Dendritic Cell Cancer Vaccine Platform
clinical development, regulatory approval and commercialization.Dr. Jenkins served as President and CEO of Dimension Therapeutics, a leading gene therapy company that she took public on NASDAQ and subsequently sold to Ultragenyx. Prior executive roles include Head of Global Research and Development
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Apr 30, 2026, 12:30 ET Northwest Biotherapeutics Appoints Dr. Annalisa Jenkins As Strategic Adviser To Advance Dendritic Cell Cancer Vaccine Platform
clinical development, regulatory approval and commercialization.Dr. Jenkins served as President and CEO of Dimension Therapeutics, a leading gene therapy company that she took public on NASDAQ and subsequently sold to Ultragenyx. Prior executive roles include Head of Global Research and Development
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Apr 30, 2026, 12:12 ET KROMATID to Present Breakthrough Genomic Integrity Benchmarking at ASGCT 2026, Powering the World's First Genomic Intelligence Platform
moment for cell and gene therapy," said Terry Opgenorth, CEO of KROMATID. "What KROMATID is building - a genomic intelligence platform – which brings clarity, context, and confidence to a space that has long operated without it. This realizes KROMATID's vision to de-risk cell and gene therapy development
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Apr 30, 2026, 11:42 ET The West Wants to Break China's Critical Minerals Stranglehold. This $68 Billion Greenland Deposit Just Locked In Three World-Class Consultants in Five Weeks.
the Skaergaard Project in Southeast Greenland — one of the largest undeveloped palladium, gold, and platinum deposits in the world — and a Cell and Gene Therapy division including Klotho's KLTO-202 program for ALS. The Company holds, through its acquisition of Greenland Mines Corp., an 80% interest
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Apr 30, 2026, 08:30 ET Factor Bioscience to Showcase Engineered iMacrophages (FACT-112) Targeting Ovarian Cancer In Vivo at ISCT 2026
Inc., a Cambridge-based biotechnology company focused on using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced its participation in the International Society for Cell & Gene Therapy (ISCT) 2026 Annual Meeting to be held in Dublin, Ireland from May
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Apr 30, 2026, 08:00 ET Genprex Receives Patent Grant from The Israel Patent Office for the Combination of Reqorsa® Gene Therapy and PD-1 Antibodies to Treat Cancer
GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that The Israel Patent Office (ILPO) has granted Genprex a patent covering the use of Reqorsa® Gene Therapy (quaratusugene ozeplasmid)
More news about: Genprex, Inc.
Apr 30, 2026, 07:47 ET NAMSA and Lexitas Announce Strategic Partnership to Deliver End-to-End Ophthalmic Medical Device Development
device-specific trial designDeep therapeutic-area coverage across anterior and posterior segment ophthalmic indications, including cell and gene therapy, rare disease and advanced modalitiesGlobal reach across the U.S., Europe, and Asia-PacificA single, accountable
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Apr 30, 2026, 07:00 ET Insmed to Present at May 2026 Investor Conferences
therapies to treat chronic, debilitating lung diseases. The Company's early-stage programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.Headquartered in Bridgewater, New Jersey,
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Apr 29, 2026, 23:13 ET D3 Bio Presents KRAS Pipeline Updates at AACR 2026; Elisrasib (D3S-001), a Next-Generation KRAS G12C Inhibitor, Shows Strong Phase 2 Efficacy Across Multiple Tumor Types
of Medicine, Korea, and lead study investigator. "Among patients whose disease progressed on first-generation inhibitors, we found five cases of KRAS gene amplification, an important mechanism of evasion of KRAS G12C inhibitor efficacy. Out of those five KRAS amplification cases, four experienced tumor
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Apr 29, 2026, 17:15 ET Denecimig (Mim8) significantly reduced annualized bleeding rate in people with hemophilia A, regardless of inhibitor status, in phase 3 data published in NEJM
factor VIIIa mimetic. J Thromb Haemost. 2024;22(4): 990-1000. doi: 10.1016/j.jtha.2023.12.016U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed August 2025. Available at
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Apr 29, 2026, 17:07 ET Denecimig (Mim8) significantly reduced annualized bleeding rate in people with hemophilia A, regardless of inhibitor status, in phase 3 data published in NEJM
factor VIIIa mimetic. J Thromb Haemost. 2024;22(4): 990-1000. doi: 10.1016/j.jtha.2023.12.016U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed August 2025. Available at
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Apr 29, 2026, 11:30 ET Industry Leaders in Fixed-Income Analytics, Kelli Sayres and Gene Park, join Andrew Davidson & Co., Inc.
Gene Park, former Managing Partners at PolyPaths, have joined the Business Development team at the 34-year-old firm.
More news about: Andrew Davidson & Co., Inc. (AD&Co)
Apr 29, 2026, 09:15 ET Expression Therapeutics Receives FDA Fast Track and Rare Pediatric Disease Designations for Investigational Stem Cell Therapy for Hemophilia A
hemophilia and cutting-edge treatment strategies for blood and immune diseases, including gene and cellular therapies, we are excited to see this innovative approach using autologous hematopoietic stem cell-based gene therapy in hemophilia A," said Glaivy Batsuli, M.D., Pediatric Hematologist/Bleeding Disorder
More news about: Expression Therapeutics, Inc.
Apr 29, 2026, 09:00 ET Kytopen, T-CURX, and the Fraunhofer IZI Establish Center of Excellence to Develop Advanced, Non-viral Cell Therapies
therapies.T-CURX, a clinical-stage biotech company developing personalized immunotherapies, will contribute its expertise and proprietary transposon-based gene transfer technology.Process development through manufacturing scale-up will be led by teams at Fraunhofer IZI.The CoE aims to develop
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Apr 29, 2026, 08:56 ET NanoMosaic to Showcase the FDA/CBER AMT Designated Analytics Platform at ASGCT 2026
/PRNewswire/ -- NanoMosaic, Inc., a leader in advanced analytical technologies for cell and gene therapy development and manufacturing, today announced its strong scientific and commercial presence at the American Society of Gene & Cell Therapy Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts.
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Apr 29, 2026, 05:00 ET Biohub Launches the Virtual Biology Initiative to Galvanize a Global Effort to Create the Open Data Foundation for AI-Accelerated Biology
diseases."The Billion Cells Project is a landmark single-cell sequencing project to advance researchers' understanding of cellular behavior and gene function. Launched in 2025 to break through the data bottleneck limiting biological AI models, the Billion Cells Project coordinates 17 projects across
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Apr 28, 2026, 16:05 ET PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam
disorder of the central nervous system.1 It is caused by a defective gene. This gene produces a protein, called Huntingtin (HTT), which is involved in the functioning of the nerve cells in the brain (neurons). When the gene is defective, it produces an abnormal (or mutated) HTT protein that is toxic
More news about: PTC Therapeutics, Inc.
Apr 28, 2026, 12:21 ET CIRM Awards AcuraStem $7.5 Million in Grant Funding to Advance AS-241 Toward a First-in-Human Trial for ALS and FTD
consequence of ALS and FTD driven by dysfunction of the protein TDP–43. Pathological loss of nuclear TDP–43 leads to cryptic splicing of the UNC13A gene, resulting in loss of a protein that is essential for healthy synaptic communication. TDP-43 pathology is observed in approximately 97% of ALS patients,
More news about: AcuraStem - Patient-Based Therapeutics