Disease, Hospitals, Therapies and Trials

SynaptixBio and Evotec extend rare disease therapies partnership

Drug Discovery World

JULY 24, 2024

The drug research was originally carried out by the Children’s Hospital of Philadelphia (CHOP), the world’s leading centre for leukodystrophy research, under a sponsored research agreement. CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC.

Therapies

Therapies Disease Clinical Trials Hospitals

First use of CAR-T therapy in patient with stiff-person syndrome

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

Therapies Disease Hospitals Treatment

First gene therapy trial for Hunter syndrome opens in Manchester, UK

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

Therapies Trials Clinical Trials Hospitals

FDA Orphan Drug Designation awarded to rare disease therapies

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.

Disease

Disease FDA Therapies Drugs

AI digital twins could replace control arm in clinical trials

Drug Discovery World

JUNE 25, 2024

A new study demonstrates that AI-powered digital twin solutions can potentially replace the standard-of-care (SOC) control arm of a clinical trial. 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.

Clinical Trials

Clinical Trials Trials Hospitals Clinical Research

FDA approves first gene therapies for sickle cell disease (with a warning)

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

FDA Approval Therapies FDA Disease

FDA approves first gene therapy for Duchenne muscular dystrophy

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

FDA Approval Therapies FDA Protein Expression

Research reveals therapeutic potential of mRNA in rare diseases

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

Disease Research RNA Therapies

Single-course in vivo base editing therapy proven to lower cholesterol

Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

Therapies

Therapies Trials Clinical Trials Disease

Amgen reports positive results for treatment of IgG4-RD

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

Treatment Medical Schools Trials Clinical Trials

Researchers investigate genetic variants linked to paediatric diseases

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.

Disease

Disease Research Hospitals DNA

Gene therapy for ‘childhood dementia’ shows promise

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. Encouraging results An improvement in neurocognitive assessments compared with natural progression of the disease in one of the children at 18-months post-treatment.

Therapies

Therapies Trials Virus Disease

Positive early data for first soluble TCR therapy for HIV

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. Although people living with HIV can control their disease with antiretroviral therapies, lifelong treatment is needed as reservoirs of HIV infected cells persist.

Therapies

Therapies Laboratories Trials Hospitals

Results revealed for ‘practice-changing’ combination liver cancer therapy

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. According to the results of an interim analysis conducted after a median follow-up of 17.4

Therapies

Therapies Medical Schools Trials Treatment

FDA approves gene therapy for rare neurological disorder

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

FDA Approval FDA Therapies Hospitals

Potential new treatment for Crohn’s disease patients in Scotland

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

Disease Treatment Hospitals Therapies

Prostate cancer combination therapy reduces risk of death

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

Therapies Hospitals Disease Treatment

Trial sheds light on combined treatment for colorectal cancer

Drug Discovery World

SEPTEMBER 15, 2022

A new combination therapy could represent a significant advancement in the treatment of metastatic colorectal cancer. Findings from the SUNLIGHT trial have revealed that the combination of trifluridine/tipiracil plus bevacizumab showed a statistically significant improvement in overall survival compared to trifluridine/tipiracil alone.

Trials

Trials Treatment Clinical Trials Hospitals

Sequana announces first patient enrolled in heart failure trial

Drug Discovery World

JULY 12, 2023

Sequana Medical, a company targeting fluid overload in liver disease, heart failure and cancer, has enrolled the first patient in its MOJAVE study. The post Sequana announces first patient enrolled in heart failure trial appeared first on Drug Discovery World (DDW).

Trials

Trials Disease Therapies Treatment

Turning Science into Business: Inducing autophagy to treat disease

Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

Disease

Disease Science Clinical Trials Therapies

Encouraging results for RESP-X in ongoing Phase I clinical trial

Drug Discovery World

APRIL 10, 2023

RESP-X, Infex Therapeutics’ anti-virulence therapy to treat Pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients, has shown a promising interim safety and tolerability profile in its ongoing first-in-human Phase I clinical trial.

Clinical Trials

Clinical Trials Trials Laboratories Clinical Research

Treatment potential for immunotherapy-induced cytokine release syndrome

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

Treatment Clinical Trials Immune Response Therapies

This week in drug discovery (27-31 May)

Drug Discovery World

MAY 31, 2024

The selected news highlights this week report breakthrough findings from early phase research and development, mainly preclinical studies and Phase I clinical trials, including a drug that can prevent sepsis deaths, a microbiome based immuno-oncology candidate, and a plant-based therapy for diarrhoea.

Small Molecule

Small Molecule Clinical Trials Drugs Trials

Investigating potential for an oral treatment in diabetic retinopathy

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

Treatment Clinical Trials Pharmacokinetics Trials

Renaissance Pharma licenses Hu14.18 for neuroblastoma

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

Licensing Licensing Hospitals Therapies

Japan becomes first country to approve Astellas’ gastric cancer mAb

Drug Discovery World

MARCH 29, 2024

targeted therapy approved by any regulatory agency in the world. Both trials met their primary endpoint, progression-free survival (PFS), as well as a key secondary endpoint, overall survival (OS), showing statistical significance in patients treated with Vyloy plus chemotherapy compared to placebo plus chemotherapy.

Clinical Trials

Clinical Trials Trials Therapies Treatment

Cancer vaccine shows sustained improvement in survival rates

Drug Discovery World

JUNE 6, 2024

The combination therapy was also shown to reduce the risk of distant metastasis or death by 62% compared to Keytruda alone in these patients. The global Phase III clinical trial is being led by University College London Hospitals NHS Foundation Trust in the UK and will include around 1,100 people. months after initial treatment.

Vaccine

Vaccine Clinical Trials FDA Approval Research Laboratories

Combination therapy halts lung cancer tumour growth in mice

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. Phase I trial on the horizon. High levels of SMYD3 are associated with more invasive disease, increased resistance to alkylating chemotherapy and worse prognosis.

Therapies

Therapies Treatment DNA Clinical Trials

How to Prioritize Pediatric Populations in NASH Trials

PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.

Trials

Trials Clinical Trials Clinical Research Disease

Promising new drug for chronic blood clotting disorder

Drug Discovery World

JUNE 28, 2023

Takeda has revealed favourable interim results from its Phase III crossover trial evaluating the safety and efficacy of TAK-755 (recombinant ADAMTS13) replacement therapy for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP). Acute TTP events have a mortality rate of >90%, if left untreated.

Trials

Trials Clinical Trials Drugs Therapies

Advancing neurological therapies with Dr Bruce Leuchter

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

Therapies

Therapies Drug Development Disease Clinical Development

Can regenerative medicine treat heart attacks?

Drug Discovery World

JULY 31, 2023

DDW’s Reece Armstrong speaks to Hendrik Streefkerk , Chief Medical Officer and Drug Safety Officer of CellProthera about the company’s cell therapy for acute myocardial infarction (AMI) and why regenerative medicine could be key to treating cardiac diseases. million people every year.

Clinical Trials

Clinical Trials Hospitals Therapies Trials

Zebrafish for Disease Modeling and Drug Screening in Pediatric Cancers

biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

Disease

Disease Medical Schools Pharma Companies Clinical Trials

LDL cholesterol vaccine ‘could be a game-changer’

Drug Discovery World

MARCH 27, 2023

The first subjects have been dosed in a Phase I clinical trial of VXX-401, an investigational vaccine designed to lower low-density lipoprotein (LDL) cholesterol, a known factor in heart disease. We’re excited to get this first-in-human trial of VXX-401 started.

Vaccine

Vaccine Immune Response Trials Clinical Trials

Six scientists named as new core institute members at Broad

Broad Institute

AUGUST 7, 2024

Core institute members have a major research presence at Broad, and remain full members of their home institution (MIT, Harvard University, or one of Harvard’s primary teaching hospitals). Anna Greka ’s research focuses on fundamental mechanisms of cellular dysfunction in genetic diseases with a focus on membrane proteins.

Medical Schools

Medical Schools Hospitals Bioinformatics Vaccine

Insights into cellular therapies for cancer treatment

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

Therapies Treatment FDA Approval Vaccine

Immutep reports positive final data at ASCO 2023

Drug Discovery World

JUNE 12, 2023

Immutep revealed positive final data from Part C of the TACTI-002 Phase II trial at the ASCO 2023 Annual Meeting in Chicago, US. The combination treatment was investigated in patients unselected for PD-L1 expression (N=37), with disease progression on, or after, platinum-based therapy (± cetuximab).

Trials

Trials Hospitals Therapies Treatment

Relieve Caregiver Burden, Improve Neurodegenerative Clinical Trials

PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

Clinical Trials

Clinical Trials Trials Clinical Research Doctors

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

The Pharma Data

DECEMBER 29, 2020

29, 2020 /PRNewswire/ — Phase 3 program in hospitalized patients to continue based on passing futility analysis on ability to reduce incidence of death or mechanical ventilation. As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes.

Hospitals

Hospitals Trials Clinical Trials Production

Five key studies into drugs for childhood cancers

Drug Discovery World

SEPTEMBER 7, 2023

Over 100 subtypes of childhood cancer exist today and it represents the most common cause of disease-related death in children. 4 Recent regulatory initiatives in the United States and Europe have aimed to increase the study of novel anti-cancer therapies in children.

Clinical Trials

Clinical Trials Trials Drugs DNA

Advancing paediatric cancer research through partnerships

Drug Discovery World

DECEMBER 28, 2023

When I was a second-year resident training as a paediatric oncologist at Nicklaus Children’s Hospital, I met a teenage boy diagnosed with Hodgkin’s Lymphoma. He was admitted to the hospital for weekly chemotherapy. When I made afternoon patient rounds, I would find him sitting up in his hospital bed doing homework.

Research

Research Clinical Trials Hospitals Trials

SARS-CoV-2 antigen levels could predict patient outcomes

Drug Discovery World

AUGUST 31, 2022

A new study, published in the Annals of Internal Medicine , shows that higher levels of viral antigen in the blood, which could indicate ongoing SARS-CoV-2 replication, correlated with more severe disease. . Analysis of Covid patient blood samples.

Hospitals

Hospitals Trials Virus International

New post-Covid study links infection and retrovirus

Drug Discovery World

SEPTEMBER 13, 2023

The first months of the trial have already confirmed that the W-ENV protein is present in the blood of over 25% of patients with persistent syndromes who suffered from Long Covid.

Protein Expression

Protein Expression Clinical Trials Hospitals Trials

UK MHRA expands Cosentyx use in paediatric arthritic conditions

Drug Discovery World

NOVEMBER 10, 2022

The drug is approved alone or in combination with methotrexate (MTX) in patients six years and older whose disease has responded inadequately to, or who cannot tolerate, conventional therapy. . ERA and JPsA are forms of JIA and are progressive, debilitating autoimmune diseases, each accounting for up to 11% of all JIA cases. .

Hospitals

Hospitals Therapies Disease Trials

SynaptixBio and Evotec extend rare disease therapies partnership

First use of CAR-T therapy in patient with stiff-person syndrome

Trending Sources

First gene therapy trial for Hunter syndrome opens in Manchester, UK

FDA Orphan Drug Designation awarded to rare disease therapies

AI digital twins could replace control arm in clinical trials

FDA approves first gene therapies for sickle cell disease (with a warning)

FDA approves first gene therapy for Duchenne muscular dystrophy

Research reveals therapeutic potential of mRNA in rare diseases

Single-course in vivo base editing therapy proven to lower cholesterol

Amgen reports positive results for treatment of IgG4-RD

Researchers investigate genetic variants linked to paediatric diseases

Gene therapy for ‘childhood dementia’ shows promise

Positive early data for first soluble TCR therapy for HIV

Results revealed for ‘practice-changing’ combination liver cancer therapy

FDA approves gene therapy for rare neurological disorder

Potential new treatment for Crohn’s disease patients in Scotland

Prostate cancer combination therapy reduces risk of death

Trial sheds light on combined treatment for colorectal cancer

Sequana announces first patient enrolled in heart failure trial

Turning Science into Business: Inducing autophagy to treat disease

Encouraging results for RESP-X in ongoing Phase I clinical trial

Treatment potential for immunotherapy-induced cytokine release syndrome

This week in drug discovery (27-31 May)

Investigating potential for an oral treatment in diabetic retinopathy

Renaissance Pharma licenses Hu14.18 for neuroblastoma

Japan becomes first country to approve Astellas’ gastric cancer mAb

Cancer vaccine shows sustained improvement in survival rates

Combination therapy halts lung cancer tumour growth in mice

How to Prioritize Pediatric Populations in NASH Trials

Promising new drug for chronic blood clotting disorder

Advancing neurological therapies with Dr Bruce Leuchter

Can regenerative medicine treat heart attacks?

Zebrafish for Disease Modeling and Drug Screening in Pediatric Cancers

LDL cholesterol vaccine ‘could be a game-changer’

Six scientists named as new core institute members at Broad

Insights into cellular therapies for cancer treatment

Immutep reports positive final data at ASCO 2023

Relieve Caregiver Burden, Improve Neurodegenerative Clinical Trials

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

Five key studies into drugs for childhood cancers

Advancing paediatric cancer research through partnerships

SARS-CoV-2 antigen levels could predict patient outcomes

New post-Covid study links infection and retrovirus

UK MHRA expands Cosentyx use in paediatric arthritic conditions

Stay Connected