Disease, Hospitals and Therapies - Drug Discovery Digest

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

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.

FDA

FDA Disease Therapies Drugs

Successful off-label use of CAR-T therapy in a child with lupus

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

Therapies Disease Treatment Hospitals

Infectious diseases research fund will progress antibody therapies

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

Disease Therapies Research Hospitals

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

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

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

Reflections from Advanced Therapies Week 2023

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

Therapies Small Molecule Production Treatment

Researchers design genetic therapy to prevent brain deterioration

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

Therapies Research Doctors Hospitals

CAR T therapy successfully treats solid tumours in China

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. However, both patients maintained a tumour-free status during long-term follow-up after receiving a combination of local and CAR T cell therapies.

Therapies

Therapies Hospitals Virus Treatment

Focus on rare disease treatments to beat kidney failure, says study

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.

Disease

Disease Treatment Hospitals Therapies

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

How ready are we for radioligand therapies?

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. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

Therapies

Therapies Treatment Regulations Production

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

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

Commercialising advanced therapies: Industry perspectives

Drug Discovery World

AUGUST 4, 2023

Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world. Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world.

Therapies

Therapies Pharma Companies Treatment Marketing

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

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

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

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). ” Amgen is now planning to file for approval in the US following results on the MITIGATE study. .”

Treatment

Treatment Medical Schools Trials Clinical Trials

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

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

Therapies Trials Clinical Trials Disease

MHRA approves drug for lung cancer with limited therapy options

Drug Discovery World

NOVEMBER 6, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised Krazati (adagrasib) to treat patients whose disease is advanced or has spread to other parts of the body, and where previous treatments have failed. More than 43,000 people are diagnosed with lung cancer every year in the UK. .

Therapies

Therapies Drugs Hospitals Treatment

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

Scientists link certain gut bacteria to lower heart disease risk

Broad Institute

APRIL 2, 2024

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels.

Disease

Disease Medical Schools Hospitals DNA

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. But it’s now known that if it stops working this can lead to many different diseases. What is autophagy?

Disease

Disease Science Clinical Trials Therapies

Why genomic healthcare data matters in the development of new therapies

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

Therapies DNA Disease Research

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

Beyond the Lab: Cell & Gene Therapy

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

Therapies Disease Clinical Development Hospitals

AI digital twins could replace control arm in clinical trials

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

Clinical Trials Trials Hospitals Clinical Research

Junk DNA: How the dark genome is changing RNA therapies

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

RNA DNA Therapies Small Molecule

From Nanotechnology to Gene Therapy: Innovations in Drug Delivery

DrugBank

JUNE 13, 2024

Similarly, for cardiovascular diseases, implantable drug-eluting stents can deliver controlled doses of anti-proliferative drugs directly to the site of arterial blockage, preventing restenosis (re-narrowing) and improving long-term integrity of the vessel.

Therapies

Therapies Drugs DNA Engineering

With retinal images and genetic data, researchers predict cardiovascular, metabolic, and other disease risks

Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

Disease

Disease Research Medical Schools Science

Top five breakthroughs in advancing breast cancer therapies

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

Therapies Small Molecule Immune Response Engineering

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. High levels of SMYD3 are associated with more invasive disease, increased resistance to alkylating chemotherapy and worse prognosis. This has not changed for 30 years.

Therapies

Therapies Treatment DNA Clinical Trials

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

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

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

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

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

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

Recent developments in on-demand voiding therapies [Minireview]

ASPET

APRIL 19, 2024

People with neurological injury (spinal cord injury, traumatic brain injury, stroke) or disease (multiple sclerosis, Parkinson's disease, spina bifida) and many elderly are unable to voluntarily initiate voiding. One cannot survive without regularly urinating and defecating.

Therapies

Therapies Hospitals Disease Drugs

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

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

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. Moitreyee Chatterjee-Kishore, Senior Vice President and Head of Immuno-Oncology Development, Astellas, said: “The approval of Vyloy by the MHLW marks a new era in the treatment of gastric cancer, offering the first and only targeted therapy option for CLDN18.2-positive

Clinical Trials

Clinical Trials Trials Therapies Treatment

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

FDA Orphan Drug Designation awarded to rare disease therapies

Trending Sources

Successful off-label use of CAR-T therapy in a child with lupus

Infectious diseases research fund will progress antibody therapies

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

Research reveals therapeutic potential of mRNA in rare diseases

FDA approves first gene therapy for Duchenne muscular dystrophy

Reflections from Advanced Therapies Week 2023

Researchers design genetic therapy to prevent brain deterioration

CAR T therapy successfully treats solid tumours in China

Focus on rare disease treatments to beat kidney failure, says study

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

How ready are we for radioligand therapies?

Researchers investigate genetic variants linked to paediatric diseases

Prostate cancer combination therapy reduces risk of death

Commercialising advanced therapies: Industry perspectives

FDA approves gene therapy for rare neurological disorder

Gene therapy for ‘childhood dementia’ shows promise

Potential new treatment for Crohn’s disease patients in Scotland

Amgen reports positive results for treatment of IgG4-RD

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

MHRA approves drug for lung cancer with limited therapy options

Positive early data for first soluble TCR therapy for HIV

Scientists link certain gut bacteria to lower heart disease risk

Turning Science into Business: Inducing autophagy to treat disease

Why genomic healthcare data matters in the development of new therapies

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

Beyond the Lab: Cell & Gene Therapy

AI digital twins could replace control arm in clinical trials

Junk DNA: How the dark genome is changing RNA therapies

From Nanotechnology to Gene Therapy: Innovations in Drug Delivery

With retinal images and genetic data, researchers predict cardiovascular, metabolic, and other disease risks

Top five breakthroughs in advancing breast cancer therapies

Combination therapy halts lung cancer tumour growth in mice

Advancing neurological therapies with Dr Bruce Leuchter

Zebrafish for Disease Modeling and Drug Screening in Pediatric Cancers

Treatment potential for immunotherapy-induced cytokine release syndrome

Insights into cellular therapies for cancer treatment

Renaissance Pharma licenses Hu14.18 for neuroblastoma

This week in drug discovery (27-31 May)

Recent developments in on-demand voiding therapies [Minireview]

Investigating potential for an oral treatment in diabetic retinopathy

Cancer vaccine shows sustained improvement in survival rates

Japan becomes first country to approve Astellas’ gastric cancer mAb

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