Drug Discovery World

AUGUST 19, 2024

The discovery of a new regulator affecting immune cells could lead to new treatments to reduce inflammation in diseases including arthritis and severe Covid-19. This activity triggers responses which are finely balanced, to protect against disease and infection, and to reduce cell-damaging inflammation.

Disease 162

Disease Drugs Regulations Research 162

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.”

Therapies 130

Therapies Disease Clinical Trials Treatment 130

Drug Discovery World

FEBRUARY 8, 2024

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s therapies are based on its antigen-specific immune tolerance platform and are designed to reprogramme the immune system to address the underlying root cause of immune-mediated diseases.

Disease 130

Disease Therapies Pharmaceuticals Clinical Trials 130

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). The drug is recommended for use on the NHS under the terms of a confidential commercial agreement.

Disease 130

Disease Therapies Treatment Drugs 130

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

Therapies Disease Engineering Medical Schools 114

Drug Discovery World

AUGUST 30, 2024

NS: Vittoria’s lead programme, VIPER-101, targeting T-cell lymphoma, reached a significant milestone with the acceptance of our Investigational New Drug (IND) application in October 2023. Beyond VIPER-101, we’re developing subsequent programmes for autoimmune disease and solid tumours.

Therapies 147

Therapies Treatment Disease Clinical Trials 147

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.” .

Disease 214

Disease Therapies Virus Treatment 214

Drug Discovery World

AUGUST 5, 2024

Theriva Biologics’ VCN-01 for retinoblastoma has been granted rare paediatric drug designation (RPDD) by the US Food and Drug Administration (FDA). VCN-01 is a systemic, selective, stroma-degrading oncolytic adenovirus, which previously received orphan drug designation for the same indication.

FDA 147

FDA Therapies Drugs Clinical Trials 147

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.

Therapies 130

Therapies Disease Clinical Trials Trials 130

Drug Discovery World

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

Engineering 284

Engineering Therapies Trials Medical Schools 284

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).

FDA 130

FDA Disease Therapies Clinical Trials 130

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.”

Therapies 130

Therapies Immune Response Disease Treatment 130

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.”

Therapies 246

Therapies Disease Hospitals Treatment 246

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.

Therapies 130

Therapies Disease Research Immune Response 130

Drug Discovery World

SEPTEMBER 9, 2024

The US Food and Drug Administration (FDA) has granted fast track designation to SYNCAR-001 + STK-009. The fast-track designation follows investigational new drug (IND) clearance for a Phase I study evaluating SYNCAR-001 + STK-009 for non-renal SLE and lupus nephritis (LN).

FDA 147

FDA Therapies Clinical Trials Engineering 147

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.

Therapies 147

Therapies DNA Disease RNA 147

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

FDA Approval 147

FDA Approval FDA Disease Therapies 147

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. LifeArc will invest £2.7 ($3.4)

Disease 130

Disease Therapies Research Hospitals 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).

FDA Approval 130

FDA Approval Therapies FDA Disease 130

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

Therapies Treatment Protein Expression Trials 312

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. We continue to make great progress with our troculeucel clinical programme in AD,” said Paul Song, Chairman and CEO of NKGen. “In

Therapies 130

Therapies Trials Treatment Disease 130

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: How does SNK01 work? RA: What are some of the unmet needs in current treatments for Alzheimer’s?

Therapies 306

Therapies Trials Treatment Disease 306

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. The post EMA prioritises AAV gene therapy for Krabbe disease appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Disease Virus Trials 130

Drug Discovery World

JUNE 9, 2023

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to DTx Pharma’s DTx-1252, an investigational small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). “We are pleased to receive Orphan Drug Designation from the FDA for DTx-1252.

Disease 130

Disease Therapies FDA RNA 130

Drug Discovery World

APRIL 23, 2024

Michael Wyand, PhD, Chief Executive Officer/Director, Oxeia Biopharmaceuticals explains why there’s a need for new drugs to treat traumatic brain injuries. Over the past 20 years research has increasingly linked concussions to neurogenerative conditions, including dementia, Alzheimer’s disease and CTE, a degenerative brain disease.

Therapies 147

Therapies Drugs Laboratories Medical Schools 147

Drug Discovery World

MARCH 8, 2023

If successful, GNT-0003 would be the first gene therapy for Crigler-Najjar syndrome.” PRIME status was granted to GNT-0003 following promising results from early phases of the European trial currently underway in collaboration with the CureCN consortium, and sponsored by Genethon.

Therapies 130

Therapies Disease Clinical Trials Trials 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.

Therapies 102

Therapies Disease RNA Engineering 102

Drug Discovery World

JANUARY 10, 2023

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for VAX-24, Vaxcyte’s 24-valent pneumococcal conjugate vaccine for the prevention of invasive pneumococcal disease. . The post Breakthrough Therapy designation for pneumococcal disease vaccine appeared first on Drug Discovery World (DDW).

Vaccine 130

Vaccine Therapies Disease Immune Response 130

Drug Discovery World

JANUARY 27, 2023

This comes just as Myosana has appointed its new CEO, Dr Matthew Lumley, a physician scientist who has spent nearly two decades in academic medicine and drug development. As proof of principle for the platform, success in treating Duchenne would open up opportunities for Myosana to target a large range of neuromuscular and cardiac diseases.”

Therapies 130

Therapies Disease Drug Development Drugs 130

Drug Discovery World

MARCH 29, 2023

Sonoma Biotherapeutics and Regeneron Pharmaceuticals have announced plans to collaborate on the discovery, development and commercialisation of novel regulatory T cell (Treg) therapies for autoimmune diseases. Sonoma will also retain full ownership of its lead cell therapy candidate, SBT-77-7101, and other programmes in development.

Therapies 130

Therapies Disease Pharmaceuticals Production 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.

Therapies 147

Therapies Bioinformatics DNA FDA 147

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.

Therapies 147

Therapies FDA Clinical Research Marketing 147

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). It is estimated that this includes around 2,000 patients in the UK. It is estimated that this includes around 2,000 patients in the UK.

Therapies 277

Therapies Clinical Trials Trials Treatment 277

Drug Discovery World

SEPTEMBER 11, 2024

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The gene therapy itself caused mild inflammation that was treated with steroids. Side effects were largely limited to minor surgical complications.

Therapies 147

Therapies Trials Clinical Trials Laboratories 147

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.

Therapies 147

Therapies Clinical Trials Trials FDA Approval 147

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.

Therapies 147

Therapies Clinical Trials FDA Clinical Development 147

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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