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.

FDA Approval 130

FDA Approval Therapies FDA Disease 130

Drug Discovery World

SEPTEMBER 25, 2024

Mylotarg was ahead of its time in many ways: its novel structure of an antibody to target diseased cells, plus a cancer-killing payload attached via a special linker, is the blueprint for the antibody-drug conjugates (ADCs) that followed.

Therapies 162

Therapies Pharma Companies FDA Approval FDA 162

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

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 147

Treatment Medical Schools Trials Clinical Trials 147

Drug Discovery World

OCTOBER 10, 2022

A new clinical trial, led by the University of Glasgow and NHS Greater Glasgow and Clyde (NHSGGC), hopes to improve treatment options for patients with Crohn’s disease. . Crohn’s disease is a chronic condition which currently affects more than 115,000 people in the UK and causes inflammation of the lining of the digestive system.

Disease 130

Disease Treatment Hospitals Therapies 130

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.

Therapies 130

Therapies Disease Treatment Hospitals 130

Drug Discovery World

JANUARY 18, 2024

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

Treatment 162

Treatment Clinical Trials Immune Response Therapies 162

Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

Disease 147

Disease Research RNA Therapies 147

Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

FDA Approval 243

FDA Approval Therapies FDA Protein Expression 243

Drug Discovery World

JANUARY 23, 2023

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

Therapies 246

Therapies Small Molecule Production Treatment 246

Drug Discovery World

OCTOBER 17, 2023

CARsgen has published evidence of over seven years of disease-free survival in advanced hepatocellular carcinoma (HCC) following treatment with its CAR-GPC3 T cell therapy. For HCC patients with concurrent inferior vena cava tumour thrombus, treatment options are limited and overall prognosis is poor.

Therapies 162

Therapies Hospitals Virus Treatment 162

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.

Therapies 147

Therapies Research Doctors Hospitals 147

Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

Treatment 130

Treatment Clinical Trials Pharmacokinetics Trials 130

Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

Drug Discovery World

FEBRUARY 28, 2023

The combination therapy of niraparib and abiraterone acetate (AA) could become the first dual action tablet (DAT) available in the European Union for metastatic castration-resistant prostate cancer (mCRPC) with BRCA1/2 mutations. A lethal disease Prostate cancer is the most common cancer in men in Europe.

Therapies 130

Therapies Hospitals Disease Treatment 130

Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline.

FDA Approval 130

FDA Approval FDA Therapies Hospitals 130

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.

Therapies 130

Therapies Trials Clinical Trials Hospitals 130

Drug Discovery World

JUNE 19, 2024

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. Its unique mechanism of action aims to deliver treatment directly to cancer cells, minimising damage to nearby healthy cells 2,5.

Therapies 147

Therapies Treatment Regulations Production 147

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

Therapies 64

Therapies Treatment FDA Approval Vaccine 64

Drug Discovery World

AUGUST 4, 2023

Dr Christian K Schneider , Head of Biopharma Excellence at PharmaLex, presents the findings of an industry panel debate on barriers to commercialisation of novel treatments, and the most effective strategies to bring them to market. Other challenges involve talent gaps, particularly now that cell and gene therapy manufacturing is maturing.

Therapies 130

Therapies Pharma Companies Marketing Treatment 130

Drug Discovery World

NOVEMBER 6, 2023

The UK regulator has approved a new treatment for non-small cell lung cancer (NSCLC) in adults that has a mutation and produces a rare protein called KRAS G12C. “The expansion of treatment options for NSCLC benefits patients and clinicians alike.

Therapies 130

Therapies Drugs Hospitals Treatment 130

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Three additional patients are currently within the normal cognitive development range at nine to 18 months post-treatment, but require longer follow-up to assess outcomes.

Therapies 130

Therapies Trials Virus Disease 130

Drug Discovery World

NOVEMBER 14, 2023

The trial participants have heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterised by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). FH is one of the most common genetic conditions, affecting around one in 300 people globally.

Therapies 130

Therapies Trials Clinical Trials Disease 130

Drug Discovery World

FEBRUARY 24, 2023

A Phase I safety and pharmacodynamic activity trial shows IMC-M113V, the first soluble T cell receptor (TCR) therapy for people living with HIV, is well tolerated. The bispecific soluble TCR therapy built on the company’s ImmTAX technology which is being developed for the treatment of people living with HIV (PLWH).

Therapies 130

Therapies Laboratories Trials Hospitals 130

Drug Discovery World

APRIL 16, 2023

Adjuvant therapy with atezolizumab (Tecentriq) and bevacizumab (Avastin) increased recurrence-free survival of patients with hepatocellular carcinoma (HCC) following surgical resection or ablation, according to the results from a Phase III trial. The positive results of IMBrave050 address this huge and urgent unmet clinical need in HCC.”

Therapies 130

Therapies Medical Schools Trials Treatment 130

Drug Discovery World

AUGUST 29, 2024

The benefits of Alzheimer’s drug lecanemab (Leqembi) are too small to justify the cost of providing the treatment to NHS patients, the UK regulator has ruled. Lecanemab is for slowing progression in mild cognitive impairment or mild dementia due to Alzheimer’s disease (AD) in adults.

Regulations 147

Regulations Hospitals Licensing Licensing 147

Drug Discovery World

SEPTEMBER 15, 2022

A new combination therapy could represent a significant advancement in the treatment of metastatic colorectal cancer. CRC is the second most common cause of cancer mortality, accounting for 881,000 deaths globally in 2018, and patients with metastatic disease have a five-year survival rate of just 11%.

Trials 130

Trials Treatment Clinical Trials Hospitals 130

Drug Discovery World

JUNE 25, 2024

The paper was co-authored by analytics company Phesi and Dr Yi-Bin Chen, Director for the Blood and Marrow Transplant Program at Massachusetts General Hospital. Phesi created a digital twin for chronic graft versus host disease (cGvHD) in the primary treatment setting.

Clinical Trials 147

Clinical Trials Trials Hospitals Clinical Research 147

KIF1A

MAY 29, 2024

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. The post Sloane’s 1st ASO treatment appeared first on KIF1A.

Treatment 52

Treatment Doctors Hospitals Therapies 52

Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. WGS and Dante’s genomic reports will provide information about predisposition to genetic diseases, genetic carrier information and more.

Therapies 246

Therapies DNA Disease Research 246

Drug Discovery World

OCTOBER 21, 2022

While advances in treatment over the last few years have resulted in good overall survival rates for patients with non-metastatic disease (90% five-year survival rate in the US 1 ), outcomes are still poor in certain cancers that do not respond to standard therapies. 3D bioprinted breast cancer tumours.

Therapies 130

Therapies Small Molecule Immune Response Engineering 130

Drug Discovery World

SEPTEMBER 5, 2022

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. The problem is that these tumours respond to treatment initially, but then they come back. But the nice thing about this strategy is that it may work where current therapies have failed.

Therapies 130

Therapies Treatment DNA Clinical Trials 130

Drug Target Review

JANUARY 23, 2024

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade. Espen integrates clinical and research expertise as Director of Medical Affairs.

Treatment 122

Treatment Immune Response Vaccine Therapies 122

Drug Discovery World

AUGUST 2, 2023

Renaissance Pharma has announced its first development programme focused on Hu14.18, a humanised anti-GD2 monoclonal antibody (mAb), licensed from St Jude Children’s Research Hospital for the treatment of newly diagnosed high-risk neuroblastoma. A novel Phase II trial incorporating Hu14.18 and three-year event-free (EFS) of 73.7%.

Licensing 246

Licensing Licensing Hospitals Therapies 246

Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

Therapies 98

Therapies Disease Clinical Development Hospitals 98

Drug Discovery World

JULY 13, 2022

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC). . Many people with sickle cell disease experience recurrent painful crises which can result in hospital admission.

Treatment 130

Treatment Hospitals Disease International 130

Drug Discovery World

AUGUST 16, 2023

Therefore, it is now more critical than ever to advance the science to develop the next generation of RNA therapies, leveraging this molecule as a therapeutic modality with transformational potential. By targeting these RNAs, we can block the fibrosis process and prevent disease progression in a highly safe, effective and accessible manner.

RNA 130

RNA DNA Therapies Small Molecule 130