Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . There are currently no approved disease modifying therapies for Huntington’s Disease. Official comments.

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Drug Discovery World

AUGUST 30, 2024

EVOQ Therapeutics has received a $2 million grant from the National Institutes of Health (NIH) to advance novel therapies for autoimmune diseases. This particular award advances therapies for myelin oligodendrocyte glycoprotein (MOG) antibody disease, a rare inflammatory disorder of the central nervous system.”

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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.” The potentially curative nature of RNDP-001 for Parkinson’s disease could dramatically alter outcomes for patients with very few treatment options.”

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Drug Discovery World

JUNE 19, 2024

KYV-101, a fully human anti-CD19 chimeric antigen receptor (CAR) T-cell product candidate, has been used to treat a 69-year-old patient suffering from treatment-refractory stiff-person syndrome (SPS). The therapy was given as part of a named-patient use in Germany for critically ill individuals who fail conventional therapies.

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Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. These deposits can cause progressive damage to the brain and other areas of the body. Disease symptomatology is characterised by chronic diarrhea during infancy and cataracts in late childhood.

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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

SEPTEMBER 5, 2023

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Elfabrio (pegunigalsidase alfa) as an option for treating Fabry disease (or alpha-galactosidase deficiency). Fabry disease is a rare genetic disease affecting approximately 1,150 people in England.

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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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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

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

DECEMBER 12, 2023

Bristol-Myers Squibb has received manufacturing and marketing approval in Japan for Abecma (idecabtagene vicleucel) as a third-line therapy for relapsed or refractory multiple myeloma (RRMM). We remain committed to researching and developing innovative therapies to transform patient lives with serious diseases through science.”

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Drug Discovery World

DECEMBER 6, 2022

CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity, according to the results of a new study in mice with an autoimmune disease similar to multiple sclerosis (MS). . I believe that this is a fully treatable disease, and CAR-T cells may be the way toward much better therapeutics.” .

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Drug Discovery World

MARCH 21, 2024

Focusing on rare conditions could significantly reduce the burden of kidney disease, according to a new study led by University College London (UCL) and the UK Kidney Association. The study, published in The Lancet to mark World Kidney Day on 14 March, draws on the largest rare kidney disease dataset ever created.

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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. RA: What are the clinical benefits of using the patient’s own cells as a treatment?

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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.

Therapies 114

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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, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Drug Discovery World

AUGUST 22, 2024

She discusses with DDW’s Megan Thomas the company’s lead programme; a gene therapy called ER-100. Chronic disease risk factors According to Dr Rosenzweig-Lipson, ageing is the biggest modifiable risk factor for most chronic diseases and is caused by a discrete number of biological mechanisms that we can target therapeutically.

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Drug Discovery World

AUGUST 20, 2024

Positive results from multiple in vivo studies have shown the impact of sphingosine kinase-2 (SPHK2) inhibition in various models of metabolic disease. The company says the data support the potential clinical use of opaganib for the prevention and therapy of Type 2 diabetes and other obesity-related disorders.

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Treatment Virus RNA Therapies 148

Drug Discovery World

AUGUST 11, 2023

Liverpool School of Tropical Medicine (LSTM) and LifeArc are partnering to launch a new Translational Development Fund to help tackle infectious diseases. million into the fund, which will support the progression of new technologies and treatments for emerging viral threats and neglected tropical diseases.

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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). Of these eligible patients, 28 (97%) were free of severe pain crises for at least 12 months after treatment.

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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

JANUARY 30, 2023

Register to attend Cell and gene therapy: sustaining growth and maximising opportunities, a free-to-attend DDW webinar, supported by Benchling and BPS Bioscience, on February 17, 2023 at 8am PST / 11am EST / 4pm GMT / 5pm CET. Cell and gene therapies (CGT) are transforming the way diseases are treated.

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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 148

Drug Discovery World

AUGUST 7, 2023

The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Susmita Sahoo, Associate Professor of Medicine, Cardiology at Icahn Mount Sinai has been exploring the use of exosomes in gene therapy for several years.

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Therapies Disease Research Immune Response 130

Drug Discovery World

AUGUST 8, 2024

The US Food and Drug Administration (FDA) has approved Voranigo, an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor, indicated for the treatment of patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma.

FDA Approval 148

FDA Approval FDA Therapies Treatment 148

Drug Discovery World

DECEMBER 4, 2023

Qnovia has announced positive results from its first in-human study of QN-01, an inhaled smoking cessation therapy. Smoking continues to be the leading cause of preventable death and disease, leading to almost eight million direct and indirect deaths annually.

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Therapies Treatment Pharmacokinetics Clinical Development 130

Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity. Other patients, some of whom are already in late stages of the disease, are awaiting treatment.

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Drug Discovery World

JULY 5, 2024

Amgen has reported positive topline results from its randomised, double-blind, multicentre, placebo-controlled Phase III clinical trial evaluating the efficacy and safety of its treatment for Immunoglobulin G4-related disease (IgG4-RD).

Treatment 148

Treatment Medical Schools Trials Clinical Trials 148

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease. Parvovirus gene therapy is a cutting-edge biomedical technology.

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Virus Therapies Treatment Medical Schools 130

Drug Discovery World

JULY 22, 2024

Keir Loiacono , CEO of BlueSphere Bio, discusses the goals of the company’s lead programme, the opportunities and potential of T-cell receptor (TCR) T cell therapies, and how to make oncology therapies tailored to individuals a reality. The goals of TCX-101 BlueSphere’s lead programme, TCX-101, includes two goals.

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Therapies Engineering Treatment Pharma Companies 130

Drug Discovery World

JULY 5, 2024

ASPIRE-FTD is evaluating investigational gene therapy AVB-101 in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein.

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Drug Discovery World

MARCH 14, 2023

Teitur Trophics, a biotech company developing new treatments for patients suffering from neurodegenerative diseases, has complete a €28M Series A financing. The financing was co-led by Sunstone Life Science Ventures and Sound Bioventures, with participation from new investors, Industrifonden, Innovestor’s Life Science Fund and P53 Invest.

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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 stem cells are thought to reduce the inflammation that drives the disease. The post Stem cell therapy shows promise in advanced MS appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 15, 2024

In June 2023, a patient at Universitätsklinikum Erlangen in Germany became the first child with lupus to receive CAR-T therapy. The treatment was the last resort to slow down systemic lupus erythematosus (SLE), which is a serious autoimmune disease that was attacking the patient’s body and seriously affected her ability to lead a normal life.

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Therapies Disease Treatment Hospitals 130

Drug Discovery World

AUGUST 21, 2024

Dr Andrea O’Hara, Senior Product Manager at Azenta Life Sciences asks why integration site analysis (ISA) is critical for the safety and efficacy of viral vectors for gene therapy. Cell and gene therapies have revolutionised how clinicians treat a variety of diseases including cancer, blindness, and metabolic disorders.

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Therapies Bioinformatics DNA FDA 148

Drug Discovery World

MAY 2, 2024

Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. 2023 was a record year for CGTs, with seven new CGTs approved by the US Food and Drug Administration (FDA) across a variety of different disease areas.

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Therapies FDA Clinical Research Marketing 148

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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