Drug Discovery World

SEPTEMBER 11, 2024

The Institute recently partnered with Automata to introduce high-throughput automation for their human cell model research programme.

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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). . The researchers set out to create CAR-T cells equipped to seek out and destroy the rogue T cells that cause MS. .

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Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

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Drug Discovery World

AUGUST 28, 2024

DDW’s Megan Thomas looks at a day in the life of Dr Joanne Taylor , Senior Vice President and Head of Discovery Research at Gain Therapeutics. MT: Can you provide a top line summary of your research? JT: Unfortunately, there are no approved disease modifying therapies for Parkinson’s disease, only symptomatic treatments.

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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

AUGUST 30, 2024

As we gear up for Phase I clinical trials in the first half of 2024, we’re aiming to evaluate the safety of our innovative CD5 knockout strategy enabled by our novel cell therapy and manufacturing platform. Beyond VIPER-101, we’re developing subsequent programmes for autoimmune disease and solid tumours.

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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

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

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

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.

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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 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. A large research collaboration, led by the University of Exeter’s MRC Centre for Medical Mycology, has focused on how immune cells sense their environment.

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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.

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Drug Discovery World

JANUARY 5, 2024

KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases. CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body.

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Drug Discovery World

JANUARY 23, 2024

Breast cancer was the most studied disease in 2023 for the third consecutive year, followed by solid tumours, stroke, Covid-19 and prostate cancer. Meanwhile, we have seen a marked reduction in investment into Covid-19 therapies in 2023, likely due to there being fewer available patients. Read the full report.

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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 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

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

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.

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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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Drug Discovery World

DECEMBER 1, 2022

PerkinElmer has launched ready-to-use adeno-associated virus vectors ( AAV ) detection kits to support researchers working on gene therapies for a variety of serious diseases. . The post Accelerating gene therapy research with AAV detection kits appeared first on Drug Discovery World (DDW).

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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

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.

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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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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

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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Therapies Trials Clinical Trials Clinical Development 147

Drug Discovery World

JUNE 6, 2024

With the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting , taking place from 30 May to 3 June, the drug discovery announcements have been coming thick and fast, with many new cancer therapies making significant strides forward in our ability to treat the disease.

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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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Therapies Disease RNA Engineering 102

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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Drug Discovery World

SEPTEMBER 4, 2024

Cancer Research Horizons and AstraZeneca have signed a five-year renewal of the Functional Genomics Centre, a facility dedicated to advancing drug discovery with genome-altering technologies such as CRISPR. It helps us tap into previously unknown biology and advances our understanding of cancer resistance mechanisms.

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Drug Discovery World

DECEMBER 13, 2022

PacBio, developer of sequencing solutions, has announced its HiFi sequencing technology will be used in a pilot project for the Children’s Rare Disease Cohorts Initiative (CRDC) at Boston Children’s Hospital. . As causative variants are identified by CRDC researchers, results are returned to participants.

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Drug Discovery World

DECEMBER 9, 2022

The ‘Rare Disease Moonshot’, a new initiative to boost research and development into rare and paediatric diseases, has launched at the European Health Summit. . It aims to speed up research into more than 7000 identified rare diseases, of which a staggering 95% have no approved treatment or cure. .

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Drug Discovery World

AUGUST 29, 2024

The US Parkinson’s Foundation has invested nearly $3 million for 33 grants to accelerate Parkinson’s disease (PD) research. The research grants will fund scientists conducting studies across various aspects of PD to bring forward new therapies, treatments, and ultimately a cure for the disease.

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Research Disease Treatment Therapies 130

Drug Discovery World

AUGUST 7, 2024

Shinobi Therapeutics has been awarded up to $59 million in non-dilutive grant funding by the Japanese Agency for Medical Research and Development (AMED). Diana Spencer, Senior Digital Content Editor, DDW The post Shinobi Therapeutics secures $59M grant for iPS-T cell therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 24, 2024

Fox Foundation for Parkinson’s Research (MJFF). calcium channel inhibition as a novel and disease-modifying approach for treatment of Parkinson’s disease. There is extensive literature linking calcium channels to pathology of the disease, and it has been demonstrated in preclinical experimental studies that the deletion of CaV2.3

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Drug Research Research Drugs Disease 130

Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.

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Disease Therapies Research Regulations 105

Drug Discovery World

OCTOBER 9, 2023

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. The post Non-viral gene therapy shows early efficacy in lung cancer appeared first on Drug Discovery World (DDW).

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