Rare disease-focused REGENXBIO hit the ground running in 2022 with its developmental programs for Duchenne Muscular Dystrophy and wet age-related macular degeneration. The Maryland-based company received clearance from the U.S. Food and Drug Administration to initiate a first-in-human study of its gene therapy for the rare form of muscular dystrophy.
REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy
REGENXBIO Inc. (Nasdaq: RGNX) today announced the clearance of its Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to evaluate RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne) in a first-in-human clinical trial. RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO’s proprietary NAV® AAV8 vector. REGENXBIO plans to initiate the trial in the first half of 2022.
DPHARM is the first event in the United States to question how we can disrupt the way clinical research gets done to reduce the burden to patients and physicians. DPHARM [….]
Novavax Initiates Phase 1/2 Clinical Trial of Combination Vaccine for COVID-19 and Seasonal Influenza
Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced enrollment of the first participants in a Phase 1/2 study to evaluate the safety and immunogenicity of a combination vaccine using Novavax’ seasonal influenza and COVID-19 vaccines. The clinical trial combines Novavax’ recombinant protein-based NVX-CoV2373 and NanoFlu™ vaccine candidates and patented saponin-based Matrix-M™ adjuvant in a single formulation (COVID-NanoFlu Combination Vaccine). Both NVX-CoV2373 and NanoFlu have previously demonstrated strong results as standalone vaccines in Phase 3 clinical trials.
American Gene Technologies (AGT) announced today that the Data and Safety Monitoring Board (DSMB) voted unanimously to continue AGT’s HIV cure program without modification, after safety analysis of the participant’s data revealed no adverse effects from the treatment. AGT’s Phase 1 trial of AGT103-T, a new cell and gene therapy for HIV disease, is designed to induce durable viral suppression by delivering therapeutic genes to the recipient’s immune cells. The cells are collected by leukapheresis, modified outside the body, then re-infused. Once infused, the cells are expected to durably suppress the virus at undetectable levels without the need for antiretroviral treatment.
GlycoMimetics Announces Initiation of Clinical Trial Evaluating Uproleselan in Combination With Venetoclax and Azacitidine
GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians at University of California (UC) Davis Comprehensive Cancer Center initiated dosing of the first patient in a clinical study of uproleselan combined with venetoclax and azacitidine for the treatment of older or unfit patients with treatment-naïve acute myeloid leukemia (AML). Brian A. Jonas, MD, PhD, FACP, UC Davis Division of Hematology/Oncology, is the clinical trial’s principal investigator.
Rockville’s NeoImmuneTech Announces First Clinical Trial Application Authorization Received in the EU for its Phase 2 Study of NT-I7 and Opdivo®
NeoImmuneTech, Inc., a clinical-stage T cell-focused biopharmaceutical company, today announced the first Clinical Trial Application (CTA) authorization in the European Union (EU). This authorization comes from Italy’s Agenzia Italiana Del Farmaco (AIFA) for the company’s ongoing Phase 2 study of NT-I7 (efineptakin alfa), a novel long-acting human interleukin-7 (IL-7), in combination with Bristol-Myers Squibb Company’s (NYSE: BMY) Opdivo® (nivolumab), a PD-1 blocking antibody, versus nivolumab monotherapy.
Novavax Initiates Pediatric Expansion for Phase 3 Clinical Trial of COVID-19 Vaccine GAITHERSBURG, Md., May 3, 2021 /PRNewswire/ — Novavax, Inc. (Nasdaq: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, today [….]
Arcellx Announces FDA Clearance of IND Application for ACLX-001, a Controllable Cell Therapy Utilizing the Company’s ARC-SparX Platform, for the Treatment of Multiple Myeloma
Arcellx, a privately held clinical-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for ACLX-001, an engineered cell therapy for the treatment of multiple myeloma.
Altimmune Commences Enrollment In Phase 1 Clinical Trial Of AdCOVID™ — A Needle-Free, Single-Dose Intranasal COVID-19 Vaccine Candidate
Altimmune Commences Enrollment In Phase 1 Clinical Trial Of AdCOVID™ — A Needle-Free, Single-Dose Intranasal COVID-19 Vaccine Candidate Intranasal administration targets the virus at its point of entry and in a [….]