FDA

PDS Biotech Announces New U.S. Patent Covering Technology Underlying PDS0101

02/03/2026

Patent Enhances IP Estate for Lead Asset; Combined with Anticipated Biologics Exclusivity, Market Protections Extend Over 20 Years Excerpt from the Press Release: PRINCETON, N.J., Jan. 22, 2026 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills…

Epicrispr Reports Early Clinical Activity and Favorable Safety Profile in First-in-Human Epigenetic Editing Study for FSHD

01/23/2026

Company to present at J.P. Morgan Healthcare Conference on Tuesday, Jan 13, 2026 Excerpt from the Press Release: SAN FRANCISCO–(BUSINESS WIRE)–Epicrispr, a clinical-stage company pioneering gene-modulating therapies, today reported early clinical data from its ongoing first-in-human, open-label study evaluating EPI-321, an investigational epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD). Epicrispr holds the first and only…

GT Biopharma Announces IND Submission for GTB-5550 TriKE®, a B7-H3-targeted natural killer (NK) cell engager for B7-H3 expressing solid tumor cancers

01/19/2026

GT Biopharma targets a portion of the estimated $362 billion global solid tumor market Preliminary, unaudited cash balance of approximately $7 million as of December 31, 2025 anticipated to extend cash runway into Q3 2026 Excerpt from the Press Release: SAN FRANCISCO, CALIFORNIA, Jan. 15, 2026 (GLOBE NEWSWIRE) — GT Biopharma, Inc. (the “Company”) (NASDAQ:…

Palvella Therapeutics Announces Positive Topline Results from Phase 2 TOIVA Clinical Trial of QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Cutaneous Venous Malformations, a Serious, Rare Genetic Disease with No FDA-approved Therapies

12/26/2025

73% of trial participants (11/15 participants) improved on the Overall Cutaneous Venous Malformations Investigator Global Assessment (Overall cVM-IGA) at Week 12; 67% of trial participants (10/15 participants) rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the Overall cVM-IGA at Week 12 Achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints,…

Pilatus Biosciences Announces FDA Clearance of IND Application for PLT012, a First-in-Class Anti-CD36 Metabolic Checkpoint Antibody in Solid Tumors

12/25/2025

Excerpt from the Press Release: DOVER, Del. & EPALINGES, Switzerland–(BUSINESS WIRE)–Pilatus Biosciences Inc., a biopharmaceutical company developing novel metabolic checkpoint immunotherapies for liver and gastrointestinal cancers, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for PLT012, a first-in-class anti-CD36 monoclonal antibody, to enter clinical…

Oncolytics Biotech® Aligns with FDA on Pivotal Study Design for Pelareorep in First-Line Pancreatic Cancer

12/01/2025

Company to launch only current registration trial with an immunotherapy in first-line pancreatic cancer in first half of 2026 Trial design allows flexibility to include an experimental arm with checkpoint inhibitor combination Excerpt from the Press Release: SAN DIEGO–(BUSINESS WIRE)–Oncolytics Biotech® Inc. (Nasdaq: ONCY) (“Oncolytics” or the “Company”), a clinical-stage immunotherapy company developing pelareorep, today…

MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome

11/14/2025

First Gene Therapy Trial for Angelman Syndrome -Orphan Drug Designation Granted for MVX-220 Excerpt from the Press Release: MIDDLETON, Mass., Nov. 6, 2025 /PRNewswire/ — MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the first patient has been dosed in the Phase 1/2…

Adagene Announces First Patient Dosed in Randomized Dose Optimization Cohort of the Phase 2 Study of Muzastotug (ADG126) in Combination with KEYTRUDA® (pembrolizumab) in Microsatellite Stable Colorectal Cancer

11/05/2025

Phase 2 clinical trial underway with first patient dosed in October to support a clear path to Phase 3 based on previous alignment with FDA Patients randomized to either 10 or 20 mg/kg of muzastotug, in combination with KEYTRUDA with up to 30 patients per arm Company anticipates trial completion in early 2027, and potential updates in…

Argá Medtech Announces First Enrollments in COHERENT-AF IDE Clinical Trial

09/30/2025

– Second generation pulsed field ablation system will be evaluated in paroxysmal and persistent atrial fibrillation patients to gain FDA approval – Excerpt from the Press Release: LAUSANNE, Switzerland and SAN DIEGO, Sept. 18, 2025 /PRNewswire/ — Argá Medtech, developers of the Coherent Sine-Burst Electroporation™ (CSE™) System, a next-generation pulsed field ablation (PFA) system for treating…

Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease

08/26/2025

Excerpt from the Press Release: CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of…