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.

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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. Both treatments were able to improve the calcium problems, but the genetic therapy was the most effective.

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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.

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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). The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 12, 2023

“For people with large B-cell lymphoma who do not respond to first-line treatment or have an early relapse, outcomes are often poor,” said Professor Roch Houot, Head of Haematology Department, University Hospital of Rennes, France.

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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.

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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. Disease research is carried out by the world’s leading centre for leukodystrophy studies, the Children’s Hospital of Philadelphia (CHOP), under a sponsored research agreement.

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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

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.

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Drug Discovery World

JANUARY 25, 2023

Vésale Bioscience, a company in the research and development of solutions and treatments using phage therapy for multi-resistant infections, has received €1.8 PhageDiag is a fast and user-friendly diagnostic technology, or phagogram, for personalised phage therapy.

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Drug Discovery World

AUGUST 11, 2023

iiCON is a consortium led by LSTM with core partners including LifeArc, Unilever, Evotec, Liverpool University Hospitals Foundation Trust, University of Liverpool, and Infex Therapeutics. LifeArc will also join the LSTM-led Infection Innovation Consortium iiCON, making its platform to progress antibody-based treatments available to partners.

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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. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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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

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Drug Discovery World

JULY 25, 2022

A new consortium has been launched in the UK to help drive innovation in the manufacturing of personalised cell therapies. . The consortium was launched by the Cell and Gene Therapy Catapult (CGT Catapult) and will be led by advanced therapy medicinal products (ATMP) developer Achilles Therapeutics. Official comments .

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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.

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Drug Discovery World

NOVEMBER 6, 2023

. “Fourteen percent of people living with NSCLC harbour the KRASG12C mutation yet there are limited targeted treatment options for patients with this devastating disease,” said Dr Shobhit Baijal, Consultant Medical Oncologist of The University Hospital Birmingham.

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Drug Discovery World

JUNE 20, 2024

UK researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. The team used a genetic therapy called silencing RNA, which blocks the action of the mutated NRAS in mole skin cells. The therapy was delivered directly to mole cells.

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SCIENMAG: Medicine & Health

JUNE 18, 2023

A three-year-long retrospective cohort study of a single Atlanta hospital’s patient population found transgender and gender-diverse teenagers rarely chose to discontinue gender-affirming hormone therapy, according to a study being presented Sunday at ENDO 2023, the Endocrine Society’s annual meeting in Chicago, Ill.

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Drug Discovery World

NOVEMBER 14, 2023

“Of the more than three million people with HeFH in the US and Europe, very few are currently at LDL-C goal, due in part to a care model that requires lifetime therapies. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 4, 2022

This transaction increases BioIVT’s global operations by adding new clinical collection sites, which are predominantly hospital based, focused on oncology, heme-oncology, and autoimmune cases,” said Dr Richard Haigh, BioIVT Chief Executive Officer (CEO).

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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.

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Fierce BioTech

AUGUST 31, 2023

How Continuous Safety Monitoring Can Reduce Hospital Stays for High-Risk Oncology Patients swheeler Thu, 08/31/2023 - 14:13 Fri, 10/06/2023 - 14:00 Resource Type Webinar Sridevi Rajeeve, MD Nicole Zahradka, PhD Duration 45 Minutes Cellular therapy products such as CAR-T show promising clinical efficacy in fighting cancer.

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

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KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. Sloane is an adorable 3 year old with KAND. Hello KIF1A Community! We are also paying for our housing.

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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. The post Why genomic healthcare data matters in the development of new therapies appeared first on Drug Discovery World (DDW). The origins .

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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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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.

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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. But the nice thing about this strategy is that it may work where current therapies have failed. Today, the typical patient survives less than a year and a half after diagnosis.

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Drug Discovery World

JULY 5, 2024

The trial met its primary endpoint with 87% of the patient cohort showing a reduction in the risk of flare-ups compared to the placebo therapy. The trial also met all key secondary endpoints including annualised flare rate; flare-free, treatment-free complete remission; and flare-free, corticosteroid-free complete remission.

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SCIENMAG: Medicine & Health

JUNE 30, 2023

The study involved ArteraAI, a developer of multimodal artificial intelligence-based predictive and prognostic cancer tests, and other researchers including those from University Hospitals (UH) Seidman Cancer Center. The information […]

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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. The post Junk DNA: How the dark genome is changing RNA therapies appeared first on Drug Discovery World (DDW).

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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?

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The Pharma Data

OCTOBER 20, 2020

20, 2020 — It may seem counterintuitive, but when someone with the eating disorder anorexia nervosa is hospitalized, treatment often begins by cutting calories. Now, new research suggests that those eating restrictions can be safely relaxed in the hospital. That led to shorter hospital stays, the study found. TUESDAY, Oct.

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SCIENMAG: Medicine & Health

JULY 14, 2023

CLEVELAND – A new study from University Hospitals (UH) Connor Whole Health describes the scope and integration of their music therapy program within10 UH medical centers.

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ASPET

APRIL 19, 2024

Catheter-associated urinary tract infections frequently require hospitalization, while manual bowel programs are time-consuming (1-2 hours), stigmatizing, and cause rectal pain and discomfort. Despite the severe, unmet, medical need; there is no literature regarding on-demand, rapid-onset, short-duration, drug-induced, voiding therapies.

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Drug Target Review

FEBRUARY 1, 2024

Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. All stakeholders have benefited from these innovations.

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Drug Discovery World

JANUARY 18, 2024

Market opportunity estimated at over US$1 billion Largely due to the CRS risk (which can be life threatening), administration of cancer immunotherapies, such as bispecific antibodies, is currently restricted to specialist cancer centres which limits uptake of these therapies. million cases of cancer by 2030 1,2.

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