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Dec 15, 2025, 09:05 ET Pivot Bio Announces Additions to Senior Management
During her time at Calyxt, she held several leadership roles across R&D operations and research, and she contributed to the launch of the first gene-edited food product on the market — Calyno high-oleic soybean oil. Earlier in her career, Liberatore completed postdoctoral work at the USDA Cereal
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Dec 15, 2025, 08:38 ET Chonnam National University Uncovers a New Molecular Target of Obesity
They found that RFP interacts directly with PPAR-γ, the master transcription factor (a special protein that governs adipocyte differentiation through gene regulation). By modulating PPAR-γ transcriptional activity, RFP increases the expression of fat cell-generating genes such as AP2 and adiponectin which
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Dec 15, 2025, 08:02 ET Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US
rare and complex genetic diseases, today announced the signing of a Memorandum of Understanding (MoU) to facilitate US commercial access to an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome, which was developed by Fondazione Telethon and has just received
More news about: Orphan Therapeutics Accelerator; Fondazione Telethon
Dec 15, 2025, 08:01 ET Sentynl Therapeutics Announces FDA Acceptance of CUTX-101 NDA Resubmission
Disease | ClinicalTrials.gov)About Menkes DiseaseMenkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially
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Dec 15, 2025, 08:00 ET Diakonos Oncology to Present Corporate Overview and Participate in Panel at 2026 Biotech Showcase
94102Panel ParticipationSession Title: Engineering the Future: Advances in Cell and Gene TherapiesDescription: Explore cutting-edge advances in cell and gene therapies that reshape the treatment landscape and create new investment opportunities. This session highlights
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Dec 15, 2025, 08:00 ET Immusoft Secures FDA Orphan Drug Designation for ISP-002 in MPS II, a Progressive Lysosomal Storage Disease
paradigm shift in the treatment of genetic diseases, while addressing key limitations associated with approved therapeutic approaches and investigational gene therapies."Orphan Drug Designation for ISP-002 is an important milestone for our MPS II program and further validates the potential of our engineered
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Dec 15, 2025, 08:00 ET Kenai Therapeutics Announces First Patient Dosed in Phase 1 REPLACE™ Clinical Trial of Neuron Replacement Cell Therapy RNDP-001 for Idiopathic Parkinson's Disease
neurological conditions. By leveraging a proprietary, Nobel Prize-winning iPSC platform, Kenai is developing off-the-shelf, allogeneic neuron replacement and gene-modified cell therapies designed to be disease-modifying. Kenai's lead candidate, RNDP-001, is in Phase 1 clinical development for moderate to moderate-severe
More news about: Kenai Therapeutics/BIOCOM
Dec 15, 2025, 07:00 ET Vanda Announces Submission of Biologics License Application to the FDA for Imsidolimab for the Treatment of Generalized Pustular Psoriasis
Imsidolimab inhibits IL-36 receptor signaling, addressing the deficiency in the endogenous IL-36RA regulator commonly seen in GPP patients due to IL36RN gene mutations.
More news about: Vanda Pharmaceuticals Inc.
Dec 15, 2025, 01:00 ET Piramal Pharma Solutions Demonstrates Commitment to Excellence with Outstanding Net Promoter Score
products and services, and potent solid oral drug products. PPS also offers development and manufacturing services for biologics including vaccines and gene therapies, made possible through Piramal Pharma Limited's associate company, Yapan Bio Private Limited.For more information visit:
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Dec 15, 2025, 00:49 ET Piramal Pharma Solutions Demonstrates Commitment to Excellence with Outstanding Net Promoter Score
products and services, and potent solid oral drug products. PPS also offers development and manufacturing services for biologics including vaccines and gene therapies, made possible through Piramal Pharma Limited's associate company, Yapan Bio Private Limited.For more information visit:
More news about: Piramal Pharma Solutions
Dec 12, 2025, 20:25 ET U.S. FDA approves AKEEGA® as the first precision therapy for BRCA2-mutated metastatic castration-sensitive prostate cancer with 54% reduction in disease progression vs standard of care*
the 162 patients with BRCA2 gene alteration(s) who received AKEEGA® in AMPLITUDE, 40% of patients were less than 65 years, 36% of patients were 65 years to 74 years, and 23% were 75 years and over.Of the 113 patients with BRCA gene alteration(s) who received AKEEGA®
More news about: Johnson & Johnson
Dec 12, 2025, 08:45 ET Actinium Pharmaceuticals Presents New Preclinical Data Demonstrating Potent Anti-Tumor Activity of ATNM-400 Across Multiple Breast Cancer Subtypes Including Hormone Receptor-Positive, Triple-Negative, and Tamoxifen- and HER2 Therapy-Resistant Breast Cancer Models at SABCS 2025
next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with r/r AML, which Actinium is seeking a potential
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Dec 12, 2025, 08:35 ET Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy
RAG-18RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression in muscle cells via RNAa mechanism. The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as
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Dec 11, 2025, 10:15 ET Cell Therapy Technologies Market worth $7.91 billion by 2030 | MarketsandMarkets™
the growth of the cell therapy technologies market include the increased funding and investment in cell therapy and the growing size of the cell and gene therapy pipeline. Furthermore, the growing adoption of automation in manufacturing processes enhances efficiency and scalability, and the growing demand
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Dec 11, 2025, 10:06 ET ASCAP UNWRAPS 2025 NEW CLASSIC HOLIDAY TOP 10 WITH HITS FROM MARIAH CAREY, KELLY CLARKSON, JUSTIN BIEBER, KATY PERRY, JIMMY FALLON, MEGHAN TRAINOR AND MORE
by Ray Evans and Jay Livingston (1950)"Feliz Navidad" by José Feliciano (1970)"Here Comes Santa Claus (Down Santa Claus Lane)" by Gene Autry and Oakley Haldeman (1947)"Deck the Halls" (Nat King Cole version), arranged by Nat King Cole and Leland Gillette (1960)"This
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Dec 11, 2025, 09:29 ET The PHERGuide study demonstrates the utility of circulating tumor DNA for the early detection and real-time monitoring of localized HER2+ breast cancer
treatment approach that combines elacestrant with everolimus in a broad group of patients with advanced ER+/HER2- breast cancer and mutations in the ESR1 gene.About MEDSIREstablished in 2012, MEDSIR distinguishes itself by its close collaboration with strategic partners to drive innovation
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Dec 11, 2025, 09:21 ET W1 REGEN DE MATELASER : lancement sur Kickstarter de l'appareil portatif de luminothérapie rouge le plus léger (24 g) et le plus puissant (5 100 mW/cm²)
une intervention chirurgicaleTendinite, épicondylite, syndrome rotulienInflammation et crispation de l'omoplate au bureauGêne articulaire liée au vieillissement et déclin microvasculaireDes laboratoires de récupération aux salles de séjour, la thérapie par laser
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Dec 11, 2025, 08:00 ET Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
President and Chief Executive Officer. "DMD is one of the most devastating childhood diseases. Families urgently need better options beyond steroids and gene-targeted approaches. While oncology remains our core focus, this milestone highlights (Z)-Endoxifen's potential as a platform therapy in both cancer
More news about: Atossa Therapeutics Inc
Dec 11, 2025, 08:00 ET Fondazione Carlo Erba e Kedrion: consegnati a Milano i Premi di Ricerca
emofilia.Premio Fabrizio Fabbrizzi:Michela Milani (Università San Raffaele di Milano - Telethon Institute for Gene Therapy) – per lo sviluppo di una terapia genica piastrinica in vivo mediata da vettore lentivirale per l'emofilia A.Premi Cecilia
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Dec 11, 2025, 07:30 ET TuHURA Biosciences Provides Corporate Update Following Recent Financing
CR rate and short duration of response as the two obstacles to improving clinical benefit in these patients."Dr. Bianco continued, "The VISTA gene is the only checkpoint upregulated in patients with AML, notably among high-risk AML. It has been shown that VISTA expression on leukemic blasts is
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Dec 10, 2025, 18:30 ET Astellas to Present New Clinical Data Across Its Gastrointestinal Cancers Portfolio at 2026 ASCO GI Cancers Symposium
A real-world study of claudin 18.2 association with molecular subtypes, mutations/biomarkers, immune landscapes, and gene signatures and prognostic value in pancreatic ductal adenocarcinoma
More news about: Astellas Pharma Inc.
Dec 10, 2025, 17:51 ET Die Fondazione Telethon gibt die FDA-Zulassung von Waskyra™ (Etuvetidigene Autotemcel) bekannt, einer Gentherapie zur Behandlung des Wiskott-Aldrich-Syndroms
Arzneimittelagentur für dasselbe Produkt nur wenige Wochen zuvor.Waskyra wurde im Rahmen jahrzehntelanger Forschung am San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Mailand entwickelt und stellt eine bedeutende wissenschaftliche und klinische Errungenschaft dar, die Patienten, die von dieser
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Dec 10, 2025, 17:45 ET ECOG-ACRIN and Caris Life Sciences unveil first findings from a multi-year collaboration to advance AI-powered multimodal tools for breast cancer recurrence risk stratification
(M+) data from 4,462 TAILORx tumor specimens. The expanded M+ gene expression panel includes 42 tumor genes associated with breast cancer recurrence derived from five commercially available gene assays, including the Oncotype DX (ODX) 21-gene recurrence score and a set of highly variable genes. Based on
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Dec 10, 2025, 17:38 ET QURE Investigation Reminder: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors with Significant Losses to Contact the Firm
On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington's disease, did not provide sufficient evidence to support uniQure's Biologics License Application ("BLA") submission. Specifically,
More news about: Kessler Topaz Meltzer & Check, LLP
Dec 10, 2025, 17:31 ET Pancreatic Cancer Market Set to Expand During the Forecast Period (2025-2034) as Precision Medicine and Immunotherapy Transform Treatment Landscape | DelveInsight
aimed at treating carcinomatous peritonitis and peritoneal dissemination, and an ALKBH3 inhibitor that targets disease progression through the ALKBH3 gene. Preclinical studies in pancreatic cancer patient-derived xenograft (PDX) models have demonstrated that subcutaneous administration of an ALKBH3 inhibitor
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