Drug Discovery World

AUGUST 14, 2024

The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.

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Drug Discovery World

JULY 14, 2022

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Hunter syndrome . Official comments .

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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

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. Setting a standard For CGTs to find success in the journey from clinic to bedside, everything from clinical trial design through to quality control requires standardisation.

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Fierce BioTech

AUGUST 14, 2024

Epilepsy Clinical Trials Finding an effective epilepsy treatment can be arduous. Download this brochure to delve into how IQVIA's expertise and innovative strategic approach can streamline the clinical trial process, helping to get new therapies to patients who need them sooner.

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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 Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Drug Discovery World

AUGUST 9, 2023

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted Aleta Biotherapeutics a clinical trial authorisation (CTA) to evaluate biologic ALETA-001 in a Phase I/II clinical trial in patients with B-cell malignancies who are relapsed/refractory to CD19 CAR T cell therapy.

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Drug Discovery World

APRIL 5, 2023

CureSearch for Children’s Cancer, a national non-profit with a mission to end childhood cancer by driving targeted and innovative research in an accelerated time frame, will fund a Phase I clinical trial to bring new therapies to paediatric brain cancer patients, including those with limited or no treatment options.

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Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). 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. “In

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Drug Discovery World

NOVEMBER 1, 2023

Axicabtagene ciloleucel, Kite Two indirect comparative analyses of CAR T-cell therapy axicabtagene ciloleucel vs. bispecific antibodies epcoritamab and glofitamab, for relapsed/refractory diffuse large B-Cell lymphoma, showed improved progression-free survival (PFS). Median PFS was 4.4 months versus 3.7 with docetaxel and the ORR was 26.4%

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Drug Discovery World

MARCH 4, 2024

In an early phase clinical trial, a combination of antibody-based medications targeting the immune system generated promising safety data and anti-tumour activity in individuals with advanced cancer. Both medications tested in the trial support immune responses against tumour cells. Various dosing schedules of CS1002 (0.3,

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Drug Discovery World

APRIL 14, 2023

Clinical trials are, undoubtedly, an essential part of the drug development process, providing us with valuable information about the safety and efficacy of new treatments and therapies. of participants 2 Covid-19 vaccination trials. In fact, these demographics which make up 13.8%

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Drug Discovery World

JULY 12, 2024

AB-1005 has also been awarded the innovative medicine designation, the Innovation Passport by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Parkinson’s disease.

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Antidote

NOVEMBER 9, 2023

Before any new treatment or therapy is able to be used by the patient population, it must go through the process of a clinical trial — this is the case for any new prescription drug, but also true for over-the-counter medications, medical devices, and more.

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

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Drug Discovery World

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The researchers plan to conduct a first-in-human clinical trial in the next five years.

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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.

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Drug Discovery World

AUGUST 8, 2024

Two veteran advocacy organisations, Reason for Hope and the Veteran Mental Health Leadership Coalition, have issued a joint statement urging the US Food and Drug Administration (FDA) to approve MDMA-assisted therapy for post-traumatic stress disorder (PTSD).

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Drug Discovery World

JULY 5, 2024

AviadoBio has launched its Phase I/II ASPIRE-FTD clinical trial at the Ohio State University in the US. ASPIRE-FTD is evaluating investigational gene therapy AVB-101 in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN).

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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Drug Discovery World

AUGUST 5, 2024

The FDA’s decision to grant rare paediatric drug designation to VCN-01 highlights the urgent need for new treatment options for paediatric patients with retinoblastoma,” said Steven Shallcross, Chief Executive Officer of Theriva Biologics. “We

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Drug Discovery World

AUGUST 19, 2024

CV6 Therapeutics has initiated patient dosing in Phase Ia clinical trial evaluating its investigational first-in-class, specific dUTPase inhibitor CV6-168 + infusional 5-fluorouracil (5-FU).

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Drug Discovery World

APRIL 16, 2023

Patients with treatment-naïve resectable non-small cell lung cancer (NSCLC) who received neoadjuvant durvalumab (Imfinzi) plus chemotherapy and adjuvant durvalumab monotherapy had improved event-free survival (EFS) and pathological complete response (pCR) compared with those who received neoadjuvant chemotherapy alone. Overall, 77.6%

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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. RA: What are the clinical benefits of using the patient’s own cells as a treatment?

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Drug Discovery World

JANUARY 22, 2024

The UK is cementing its position as a global leader in cell and gene therapy (CGT), according to a new report released today by the UK BioIndustry Association (BIA), in collaboration with Citeline. The UK has 84 drugs in clinical development, surpassing all other European countries, with 23 CGTs are already approved for patient use in the UK.

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Conversations in Drug Development Trends

MAY 10, 2024

By: Christine Moore, PhD, Vice President, Neuroscience, Scientific Solutions Patients with borderline personality disorder (BPD) face a heightened risk of substance abuse and suicide, as well as substantial delays in receiving treatment.

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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).

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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).

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Drug Discovery World

JULY 19, 2024

Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . There are currently no approved disease modifying therapies for Huntington’s Disease.

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Drug Discovery World

JUNE 19, 2024

New research has uncovered how different people respond to sepsis based on their genetics, which could lead to the development of targeted therapies. The ultimate aim is for patients to receive the most effective treatment for their sepsis more quickly, based on their immune response rather than their symptoms.

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Drug Discovery World

DECEMBER 4, 2023

Qnovia has announced positive results from its first in-human study of QN-01, an inhaled smoking cessation therapy. QN-01 is designed to meet the urgent need for new safe and effective pharmacotherapies that address the shortcomings of currently available nicotine replacement therapies (NRTs).

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Antidote

JULY 13, 2022

Before a potential new therapy can be administered to patients, it must go through several clinical trial phases designed to test the drug or device for safety and effectiveness. For many new treatments, it will take approximately ten years for the therapy to progress from initial discovery to being approved.

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Drug Discovery World

MAY 6, 2024

The company’s lead therapeutic peptide candidate is currently in a Phase III clinical trial for the treatment of hypoparathyroidism. Amolyt Pharma has announced that its lead therapeutic peptide candidate, eneboparatide, has been granted fast track designation by the U.S. Food and Drug Administration (FDA).

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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.

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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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