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CTIBIOTECH™ Partners with SATT AxLR and CILCARE to Develop Inner Ear Organoids for Hearing Disorder Screening

CTIBIOTECH™, a leading innovator in human tissue engineering and 3D bioprinting, is proud to announce its selection by SATT AxLR and CILCARE to support the development of the OrgaEar project. This strategic collaboration aims to create a world...

LOTUS study data on DAYBUE® (trofinetide) for Rett Syndrome's long-term efficacy and tolerability is published in Developmental Medicine and Child Neurology

Acadia Pharmaceuticals Inc. announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability...

CorrectSequence Therapeutics' CS-101 Achieves Promising Results in First Patient: High-Precision Base Editing Clinical Treatment for Sickle Cell Disease

CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, today announced the successful treatment of the first sickle...

Theravance Biopharma completes enrollment in Phase 3 CYPRESS study of Ampreloxetine for neurogenic orthostatic hypotension

Theravance Biopharma, Inc. announced completion of enrollment in the open-label portion of its pivotal Phase 3 CYPRESS study in patients with symptomatic neurogenic orthostatic hypotension (nOH) due to multiple system atrophy (MSA), a rare and...

Jazz Pharmaceuticals announces the U.S. FDA approval of Modeyso™ (dordaviprone) as the first and only treatment for recurrent H3 K27M-mutant diffuse midline glioma

Jazz Pharmaceuticals plc announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Modeyso™ (dordaviprone) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma...

Neurocrine Biosciences Presents New Post-Hoc Data from the KINECT®-HD Study, Demonstrating a Significant Reduction in Disease Burden with INGREZZA® (valbenazine) Capsules for Huntington's Disease Chorea

Neurocrine Biosciences, Inc. announced new patient-reported post-hoc data from the KINECT®-HD study highlighting significant reductions in both cognitive and motor-related burdens among adults treated for Huntington's disease chorea with...

Innovent Announces Approval from China's NMPA for Mazdutide, the First Dual GCG/GLP-1 Receptor Agonist, for Chronic Weight Management

Innovent Biologics, Inc. a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncologic, autoimmune, cardiovascular and metabolic, ophthalmologic and other major...

Emulate introduces the AVA™ Emulation System, a groundbreaking high-throughput organ-chip platform designed to accelerate drug development

Emulate, Inc., the world leader in Organ-on-a-Chip technology, today announced the commercial launch of the AVA™ Emulation System, a self-contained instrument that cultures, incubates, and images up to 96 independent Organ-Chip samples in a single...

Innorna Announces FDA Grants Rare Pediatric Disease and Orphan Drug Designations to IN013 for Wilson Disease Treatment

Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease...

Pint Pharma secures ANVISA approval for BESREMi® to treat Polycythemia Vera

Pint Pharma and PharmaEssentia announced that ANVISA (Brazilian Health Regulatory Agency) has approved BESREMi® (ropeginterferon alfa-2b) for the treatment of adult patients with Polycythemia Vera (PV). Polycythemia Vera (PV) is a rare, chronic,...

Fzata secures a clinical trial agreement for FZ002, targeting C. difficile treatment.

Fzata, Inc. announces the execution of a clinical trial agreement with the National Institutes of Health (NIH) to sponsor a Phase 1 clinical trial of FZ002 for C. difficile treatment. Oral FZ002 is a live yeast genetically modified to express a...

Shorla Oncology Announces the Launch of Dedicated Sales Force and Market Access Teams as Part of Expansion of Its U.S. Commercial Footprint

Shorla Oncology a U.S.-Ireland specialty pharmaceutical company, announced the launch of a dedicated sales force and market access teams and the commercial availability of IMKELDI, the first oral liquid form of imatinib to treat certain forms of...

NLS Pharmaceutics and Kadimastem announce a $500,000 initial closing of a $1 million fundraising, with shares priced at $3.10, a 48% premium over the January 6, 2025 market price

NLS Pharmaceutics Ltd. a Swiss clinical-stage biopharmaceutical company focused on the discovery and development of innovative therapies for rare and complex central nervous system disorders, and Kadimastem Ltd. "Kadimastem", a clinical-stage cell...

Pilatus Biosciences Inc. earns FDA Orphan Drug Designation for PLT012, targeting liver and intrahepatic bile duct cancer

Pilatus Biosciences Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its leading molecule, PLT012, for treating liver and intrahepatic bile duct cancer (HCC/ICCA). Achieved in November...

Preclinical study shows NUZ-001 reduces TDP-43 aggregation in ALS

Neurizon Therapeutics Limited a clinical-stage biotech company advancing treatments for neurodegenerative diseases, is pleased to announce positive results from a preclinical study of its lead candidate, NUZ-001. These innovative studies reveal...

GoodRx debuts a new e-commerce platform, starting with Opill®

GoodRx the leading prescription savings platform in the U.S., announced the rollout of its new e-commerce solution, with Opill® as its launch partner. This new seamless direct-to-consumer shopping experience allows consumers to order eligible...

Citius Pharmaceuticals' LYMPHIR™ wins FDA approval for relapsed/refractory cutaneous T-cell lymphoma

Citius Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved LYMPHIR™ (denileukin diftitox-cxdl), a novel immunotherapy for the treatment of r/r cutaneous T-cell lymphoma (CTCL) after at least one prior...

Teva and Sanofi Announce Accelerated Anti-TL1A Phase 2b Program for Inflammatory Bowel Disease

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. and Sanofi announced an update to the timing for the anti-TL1A, duvakitug (formerly known as TEV-’574/SAR447189) program investigating the human IgG1-λ2 monoclonal...

Transposon announces Phase 2 results for TPN-101 in treating C9orf72-related ALS and frontotemporal dementia

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer's disease, announced final results from its Phase...

Neurocrine Biosciences Launches INGREZZA® SPRINKLE (valbenazine) Capsules.

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) announced the launch of a new sprinkle formulation of INGREZZA® (valbenazine) capsules, the #1 prescribed vesicular monoamine transporter 2 (VMAT2) inhibitor indicated for the treatment of adults with...

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