Drug Discovery World

DECEMBER 12, 2023

Bristol-Myers Squibb has received manufacturing and marketing approval in Japan for Abecma (idecabtagene vicleucel) as a third-line therapy for relapsed or refractory multiple myeloma (RRMM). We remain committed to researching and developing innovative therapies to transform patient lives with serious diseases through science.”

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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 currently being evaluated in the US by the FDA’s Center for Drug Evaluation and Research (CDER) and in the UK by the Medicines and Healthcare Products Regulatory Agency (MHRA).

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Drug Discovery World

SEPTEMBER 16, 2024

NKGen Biotech has announced promising early interim data from the Phase I cohort trial of troculeucel, its cryopreserved autologous enhanced natural killer (NK) cell therapy for moderate Alzheimer’s disease. Read the DDW interview with Paul Song.

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Drug Discovery World

JUNE 19, 2024

KYV-101, a fully human anti-CD19 chimeric antigen receptor (CAR) T-cell product candidate, has been used to treat a 69-year-old patient suffering from treatment-refractory stiff-person syndrome (SPS). The therapy was given as part of a named-patient use in Germany for critically ill individuals who fail conventional therapies.

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Drug Discovery World

APRIL 21, 2023

New study results have demonstrated the potential of a new drug to reprogramme the immune profile in the tumour microenvironment (TME) and convert resistant tumours to responders to Immune Checkpoint Blockage (ICB) therapies. No such effects were detected with either therapy alone.

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Drug Discovery World

SEPTEMBER 2, 2024

The European Commission (EC) has approved Braftovi (encorafenib) in combination with Mektovi (binimetinib) for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with a BRAFV600E mutation. In the treatment-naïve population, the ORR was 75%, including 15% complete responses (CRs) and 59% partial responses (PRs).

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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

MARCH 11, 2024

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to iOnctura for its autotaxin inhibitor cambritaxestat for the treatment of pancreatic cancer. Cambritaxestat is being developed as a first-in-class therapy for highly fibrotic cancer indications.

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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. This paired with next generation sequencing technologies helping to identify potential gene therapy candidates for more effective and targeted interventions will be powerful.

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Drug Discovery World

AUGUST 20, 2024

The company says the data support the potential clinical use of opaganib for the prevention and therapy of Type 2 diabetes and other obesity-related disorders. The studies showed the benefit of opaganib therapy in suppression of HFD-induced body weight gain, loss of glucose tolerance and fat deposition.

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Drug Discovery World

JULY 29, 2024

Cell and gene therapies (CGTs) are the fastest growing area of therapeutics, with many preclinical and clinical programmes currently underway. New approaches to how advanced therapies are regulated and funded may be necessary for these treatments to reach the patients that need them.

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Drug Discovery World

AUGUST 30, 2024

EVOQ Therapeutics has received a $2 million grant from the National Institutes of Health (NIH) to advance novel therapies for autoimmune diseases. This particular award advances therapies for myelin oligodendrocyte glycoprotein (MOG) antibody disease, a rare inflammatory disorder of the central nervous system.”

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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. Focal cortical dysplasia is caused by areas of the brain that have developed abnormally and is among the most common causes of drug-resistant epilepsy in children.

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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). Of these eligible patients, 28 (97%) were free of severe pain crises for at least 12 months after treatment.

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Drug Discovery World

AUGUST 28, 2024

Under the collaboration, the companies will analyse somatic mutations that occur in diseased patient tissue to inform the discovery and development of therapies for cardiovascular and renal diseases. The collaboration was the first initiated under Flagship’s Pioneering Medicines strategic partnership with Pfizer.

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Drug Discovery World

APRIL 23, 2024

Michael Wyand, PhD, Chief Executive Officer/Director, Oxeia Biopharmaceuticals explains why there’s a need for new drugs to treat traumatic brain injuries. While those therapies provide some relief to concussed patients, 20% or more continue to have persistent symptoms for weeks to months to years after injury.

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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

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. This was a very small, early-stage study and we need further clinical trials to find out if this treatment has a beneficial effect on the condition.

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Therapies Clinical Trials Trials Treatment 264

Drug Discovery World

MARCH 4, 2024

In this first-in-human multicentre, open-label study conducted at nine study sites in Australia and China, Phase Ia involved monotherapy dose-escalation (Part 1), which was followed by Phase Ib combination therapy dose escalation (Part 2) and expansion (Part 3). Various dosing schedules of CS1002 (0.3,

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Therapies Immune Response Clinical Trials Trials 289

Drug Discovery World

AUGUST 22, 2024

She discusses with DDW’s Megan Thomas the company’s lead programme; a gene therapy called ER-100. Life Biosciences’ gene therapy approach differs from traditional gene replacement and gene editing approaches in that the company are not altering the DNA sequence.

Therapies 147

Therapies DNA Disease RNA 147

Drug Discovery World

AUGUST 8, 2024

The US Food and Drug Administration (FDA) has approved Voranigo, an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor, indicated for the treatment of patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma.

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Drug Discovery World

JANUARY 30, 2024

Puma Biotechnology’s Nerlynx is a pan HER TKI indicated for the extended adjuvant treatment of adult patients with early stage and metastatic HER2 positive breast cancer. The post Prescription drug could prevent cancer therapy-related diarrhoea appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. .

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Virus Therapies Treatment Medical Schools 130

Drug Discovery World

MAY 2, 2024

Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. 2023 was a record year for CGTs, with seven new CGTs approved by the US Food and Drug Administration (FDA) across a variety of different disease areas.

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Drug Discovery World

JULY 9, 2024

The Scottish Medicines Consortium (SMC) has published final advice on two rare disease therapies. Elfabrio (pegunigalsidase alfa), a novel enzyme replacement therapy (ERT), has been approved as a treatment option for Fabry disease in adults. Birch bark extract gel is the only licenced therapy for junctional and dystrophic EB.

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Drug Discovery World

AUGUST 19, 2024

The study will assess the safety, pharmacokinetic and pharmacodynamic profiles and anti-tumour activity for CV6-168 in the treatment of patients with advanced metastatic solid tumours refractory to standard treatment, including colon, gastric, breast, ovarian, lung, and other solid tumours.

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Drug Discovery World

JULY 8, 2024

Biopharmaceutical company Syncromune has had its lead-candidate for the treatment of metastatic castrate-resistant prostate cancer (mCRPC) granted Fast Track designation by the FDA. We look forward to initiating trials at multiple US sites later this year to expand our efforts to develop the SYNC-T SV-102 Therapy.”

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FDA Treatment Therapies Vaccine 147

Drug Discovery World

FEBRUARY 6, 2024

The first UK patients have received an mRNA cancer therapy – mRNA-4359 – as part of a Phase I/II clinical trial investigating its potential for treating melanoma, lung cancer and other solid tumour cancers. The study will assess the therapy administered alone or in combination with immune checkpoint inhibitor pembrolizumab.

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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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Drug Discovery World

JULY 5, 2024

ASPIRE-FTD is evaluating investigational gene therapy AVB-101 in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Targeted, minimally-invasive delivery AVB-101 is delivered as a one-time-only treatment using a minimally invasive, stereotactic neurosurgical procedure directly to the thalamus.

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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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FDA Therapies Clinical Trials Government 147

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

JUNE 28, 2024

A new Phase II trial, REGENERATE-PD, will study AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. The trial will include an estimated 87 subjects with study sites located in the US, UK and Europe.

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Drug Discovery World

JULY 18, 2024

CatalYm has completed a $150 million Series D financing to support the Phase II development of its cancer therapy visugromab. The proceeds will fund the expansion of the company’s broad Phase IIb development of visugromab into randomised Phase IIb studies in select checkpoint naïve frontline and second-line treatment settings.

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Drug Discovery World

SEPTEMBER 4, 2024

The European Commission has granted orphan medicinal product designation (OMPD) to Cereno Scientific’s lead drug candidate CS1 for pulmonary arterial hypertension (PAH). The EU OMPD complements the orphan drug designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets.

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Drug Discovery World

AUGUST 22, 2024

The US Food and Drug Administration (FDA) has approved lazertinib (Lazcluze) in combination with amivantamab-vmjw (Rybrevant) for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC).

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Drug Discovery World

JULY 17, 2024

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Kyverna Therapeutic’s CAR-T candidate KYV-101 for refractory stiff-person syndrome (SPS). The RMAT designation was based on positive clinical outcomes in patients treated in Germany under a named-patient treatment option.

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