Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. Parkinson’s disease is highly associated with mitochondrial dysfunction. Mission is planning to initiate a MTX325 Phase I trial in humans in early 2024.

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Drug Discovery World

JANUARY 5, 2024

Kyverna Therapeutics has revealed plans to progress to Phase II trials of KYV-101 for multiple sclerosis (MS) following a green light from the US Food and Drug Administration (FDA). KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases.

Therapies 312

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Drug Discovery World

APRIL 12, 2024

The US Food and Drug Administration (FDA) has accepted an Investigational New Drug (IND) application for Tr1X’s TRX103 for the prevention of Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched haematopoietic stem cell transplantation (HSCT). “As a result, innovative treatments are urgently needed.”

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Drug Discovery World

FEBRUARY 27, 2024

Arvinas has commenced dosing in the Phase I clinical trial of ARV-102, its first oral PROTAC (PROteolysis-TArgeting Chimera) protein degrader in development to treat neurodegenerative diseases. The post First PROTAC degrader for neurodegenerative disease enters trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 6, 2024

“Their guidance will be invaluable as we soon advance our lead candidate, RNDP-001, into the clinic for the treatment of Parkinson’s disease.” Proceeds from the financing will enable the company to submit an IND for RNDP-001 and complete Phase I clinical trials, which will initiate within the year.

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Drug Discovery World

FEBRUARY 8, 2024

The funds will be used to support the advancement of COUR’s myasthenia gravis (MG) and type 1 diabetes (T1D) product candidates into Phase IIa clinical trials. COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases.

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Drug Discovery World

AUGUST 30, 2024

Dr Nicholas Siciliano, Co-Founder of Vittoria Biotherapeutics, shares insight on the VIPER-101 trial targeting T-cell lymphoma, the company’s aim to ensure progression free survival and opportunities in this sector. Beyond VIPER-101, we’re developing subsequent programmes for autoimmune disease and solid tumours.

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Therapies Treatment Disease Clinical Trials 147

Drug Discovery World

SEPTEMBER 16, 2024

NKGen Biotech has announced promising early interim data from the Phase I cohort trial of troculeucel, its cryopreserved autologous enhanced natural killer (NK) cell therapy for moderate Alzheimer’s disease. Read the DDW interview with Paul Song.

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Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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Drug Discovery World

JUNE 19, 2024

The therapy was given as part of a named-patient use in Germany for critically ill individuals who fail conventional therapies. These dramatic improvements – if confirmed by further studies – may eventually provide renewed hope for a much-needed paradigm shift in the treatment of debilitating autoimmune diseases.”

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Drug Discovery World

SEPTEMBER 9, 2024

SYNCAR-001 + STK-009 is a cytokine-inducible, two-component therapy consisting of SYNCAR-001, an autologous CD19-targeting CAR-T which expresses an engineered IL-2 receptor allowing it to selectively receive a signal from STK-009, an engineered IL-2 cytokine.

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Drug Discovery World

FEBRUARY 21, 2024

DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?

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Therapies Trials Treatment Disease 306

Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The post FDA pilot programme to boost rare disease therapies appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 20, 2023

Cerevance has dosed the first patient in its ASCEND Phase II study to assess the efficacy and safety of CVN424 as a monotherapy in early-stage Parkinson’s disease. This study aims to build upon the promising results from our previous Phase II trial of CVN424 as an adjunctive therapy for later-stage Parkinson’s disease.

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Therapies Trials Disease Treatment 147

Drug Discovery World

MARCH 6, 2024

This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination. Dan Williams, CEO at SynaptixBio, said; “This ODD is a huge boost to our efforts in tackling these devastating, life-limiting rare diseases.

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Drug Discovery World

AUGUST 20, 2024

The US Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD). ORR was 75% in the 79 patients treated with the recommended dosage. The median time to first response was 1.5

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FDA Approval FDA Disease Therapies 147

Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Therapies Clinical Trials Trials Treatment 277

Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

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Drug Discovery World

SEPTEMBER 2, 2024

DDW’s Megan Thomas discusses the future of the cell and gene therapy (CGT) sector with industry experts, who predict what to expect in 2024 and beyond. The CGT landscape is showing the prospect of significantly advancing healthcare for hard-to-treat diseases,” says Josh Ludwig, Global Director, Commercial Operations, ScaleReady.

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Therapies Clinical Trials Trials FDA Approval 147

Drug Discovery World

SEPTEMBER 4, 2024

The first patient has been treated in a Phase II trial of EVO756 in adults with chronic inducible urticaria (CIndU). Evommune’s pre-clinical data demonstrates that by blocking activation of MRGPRX2 via all disease relevant ligand categories, EVO756 can prevent the degranulation of mast cells.

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Clinical Trials Trials Small Molecule Clinical Development 130

Drug Discovery World

MARCH 8, 2023

The manufacturer, Genethon, is a non-profit gene therapy R&D organisation founded by the French Muscular Dystrophy Association (AFM-Telethon). “We’re excited about the EMA’s PRIME recognition of GNT-0003,” said Genethon CEO Frederic Revah. “If Symptoms of the most severe form of the disease become apparent shortly after birth.

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Therapies Disease Clinical Trials Trials 130

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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Drug Discovery World

NOVEMBER 28, 2023

An international team of scientists has shown that a stem cell therapy for multiple sclerosis (MS) could prevent further damage to the brain. The early-stage clinical trial was the result of a collaboration between researchers at University of Colorado Anschutz Campus, the University of Cambridge and the University of Milano-Bicocca. “We

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Therapies Clinical Trials Trials Treatment 264

Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

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Therapies Clinical Trials Trials International 277

Drug Discovery World

JULY 12, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy.

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Therapies Clinical Trials FDA Clinical Development 147

Fierce BioTech

SEPTEMBER 5, 2024

How can they engage providers and raise awareness of gene therapies for the patients who need them most? png Listing Introduction Explore how incorporating genetic services can increase identification of patients who meet trial-specific inclusion/exclusion criteria, helping more patients benefit from life-changing therapeutics.

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Trials Disease Clinical Trials Therapies 52

Drug Discovery World

JULY 17, 2024

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Kyverna Therapeutic’s CAR-T candidate KYV-101 for refractory stiff-person syndrome (SPS). KYV-101 is an autologous, fully human CD19 CAR-T cell product candidate for use in B cell-driven autoimmune diseases.

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Drug Discovery World

FEBRUARY 15, 2023

With this financing, we expect to reach several key clinical milestones across multiple disease areas with unmet needs.” Cerevance has a growing pipeline of clinical and preclinical programs developed using the company’s NETSseq platform to discover and validate novel therapeutic targets in some of the most challenging neurological diseases.

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Drug Discovery World

JANUARY 25, 2023

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge Biologic’s adeno-associated virus (AAV) drug candidate for treating patients with Krabbe disease. A safer environment for gene replacement The designation follows positive safety and efficacy data from the ongoing Phase I/II RESKUE trial.

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Therapies Disease Virus Trials 130

Drug Discovery World

JULY 19, 2023

Eli Lilly has announced positive results of the TRAILBLAZER-ALZ 2 Phase III study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease.

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Disease Trials Clinical Trials Therapies 130

Drug Discovery World

JULY 26, 2023

ARMGO Pharma has enrolled the first patient in a Phase II proof-of-concept clinical trial using its Rycal ARM210 (also known as S48168), for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). CPVT is a rare genetic heart disease causing arrhythmia.

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Drug Discovery World

AUGUST 5, 2024

The FDA grants RPDD for rare diseases (fewer than 200,000 affected persons in the United States) that are serious and life-threatening and primarily affect children ages 18 years or younger. Theriva Biologics’ VCN-01 for retinoblastoma has been granted rare paediatric drug designation (RPDD) by the US Food and Drug Administration (FDA).

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Drug Discovery World

JULY 19, 2024

Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.

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Therapies Research Clinical Trials Science 130

Drug Discovery World

DECEMBER 21, 2022

million (US$2 million) from Innovate UK’s Biomedical Catalyst 2022 funding competition, to accelerate development of a first-in-class disease modifying therapy for Huntington’s disease. . awarded to accelerate therapy for Huntington’s disease appeared first on Drug Discovery World (DDW). The post £1.7M

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Therapies Disease Clinical Trials Treatment 130

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Our team of chemists then uses this information to develop novel therapies.

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