MedPath

Quidditas Secures €2.62M to Advance Revolutionary Genome Editing Technology for Rare Disease Treatment

5/12/2025

Belgian biotech Quidditas Therapeutics has raised €2.62M to develop its GREAT technology platform, which enables precise genetic recombination of any length at any genomic location, overcoming limitations of current CRISPR systems.

FDA Approves Teplizumab: First Disease-Modifying Drug for Type 1 Diabetes Prevention

11/20/2022

Teplizumab has received FDA approval as the world's first disease-modifying therapy for type 1 diabetes, delaying onset by an average of three years in high-risk individuals aged eight and older.

Contineum Therapeutics On Track for Key Clinical Milestones with PIPE-791 and PIPE-307 Programs

5/15/2025

Contineum Therapeutics expects topline data from its PIPE-791 Phase 1b PET trial in Q2 2025 and PIPE-307 Phase 2 VISTA trial for relapsing-remitting multiple sclerosis in H2 2025, affirming its clinical development timeline.

FDA Approves Zanidatamab (Ziihera) for HER2-Positive Biliary Tract Cancer

11/21/2024

The FDA has granted accelerated approval to zanidatamab-hrii (Ziihera) for previously treated, unresectable or metastatic HER2-positive biliary tract cancer.

FDA Grants Accelerated Approval to Zanidatamab for HER2-Positive Biliary Tract Cancer

11/21/2024

The FDA has granted accelerated approval to Ziihera (zanidatamab-hrii) for adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer.

NRx Pharmaceuticals Plans NDA Submissions for NRX-100 and NRX-101 in Suicidal Depression

11/14/2024

NRx Pharmaceuticals is on track to file NDAs for NRX-100 (IV Ketamine) for suicidal ideation in depression and NRX-101 (Oral D-Cycloserine/Lurasidone) for bipolar depression with suicidality by year-end 2024.

GH Research's 5-MeO-DMT Trial for Treatment-Resistant Depression Faces FDA Hold Amidst Development

11/15/2024

GH Research reported a net loss of $12.1 million in Q3, driven by increased R&D spending on clinical trials for its 5-MeO-DMT therapies.

GSK's Blenrep Demonstrates Significant Survival Benefit in Multiple Myeloma Trial, Paving Way for Potential Comeback

11/15/2024

GSK's Blenrep, combined with chemotherapy and a steroid, reduced the risk of death by 42% compared to a standard-of-care treatment in relapsed or refractory multiple myeloma.

Switching Between Rituximab Biosimilars Does Not Raise Adverse Event Risk, Study Finds

11/22/2024

A recent study published in *Cancers* indicates that switching between rituximab biosimilars does not elevate the risk of adverse reactions in patients with hematological diseases.

EU Approves Leqembi for Early-Stage Alzheimer's, Citing Benefit-Risk Balance

11/16/2024

The European Medicines Agency (EMA) has approved Leqembi for treating early-stage Alzheimer's disease, reversing an earlier rejection due to concerns about side effects.

BioMarin's Voxzogo Demonstrates Sustained Height Gains in Achondroplasia Patients

11/16/2024

Real-world data from the CrescNet registry showed that children treated with Voxzogo experienced an average height increase of 6.36 cm after 12 months.

Clinical Trials Offer Hope for Children with Rare Brain Cancer, ETMR

11/23/2024

Embryonal tumor with multi-layered rosettes (ETMR) is a rare, aggressive brain cancer in children, lacking a standard treatment protocol, making clinical trials a crucial option.

COVID-19 Infection Linked to Cancer Regression via Novel Immune Cell Activation

11/18/2024

Researchers have discovered that SARS-CoV-2 RNA can trigger the development of inducible nonclassical monocytes (I-NCMs), a unique type of immune cell with anti-cancer properties.

Infigratinib Shows Promise as Oral Treatment for Achondroplasia in Children

11/18/2024

A Phase II study reveals that infigratinib, an oral medication, significantly increases height in children with achondroplasia, the most common form of dwarfism.

Trastuzumab Duocarmazine Demonstrates Efficacy in HER2-Positive Metastatic Breast Cancer

11/24/2024

Trastuzumab duocarmazine (T-Duo) significantly improved progression-free survival in patients with advanced HER2-positive metastatic breast cancer compared to physician's choice of treatment.

CHMP Recommends AstraZeneca's Tagrisso for EU Approval in Advanced NSCLC

11/19/2024

The CHMP has recommended Tagrisso (osimertinib) for EU approval to treat locally advanced, unresectable non-small cell lung cancer (NSCLC) with specific EGFR mutations.

Zydus Healthcare to Conduct Phase III Trial for Glycopyrrolate/Indacaterol Inhaler

11/24/2024

Zydus Healthcare will conduct a Phase III clinical trial for its fixed-dose combination of glycopyrrolate and indacaterol metered-dose inhalation, as recommended by the CDSCO panel.

FDA Approves AI Tool icobrain aria for Detecting ARIA in Alzheimer's Immunotherapy Patients

11/19/2024

The FDA has approved icobrain aria, an AI-driven software, to assist in detecting ARIA (Amyloid-Related Imaging Abnormalities) in MRI scans of patients undergoing amyloid immunotherapy.

Injectable Oxygen Microbubbles Show Promise in Treating Hypoxemia and Preventing Organ Damage

11/25/2024

Researchers have developed injectable oxygen microbubbles for rapid oxygen delivery during critical moments of cardiac or respiratory arrest, addressing the limitations of traditional methods.

Zerlasiran Shows Significant Lipoprotein(a) Reduction in Phase 2 Trial

11/20/2024

Zerlasiran, a novel siRNA, significantly reduced time-averaged lipoprotein(a) levels by over 80% in a Phase 2 trial, offering a potential treatment for elevated Lp(a).

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