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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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

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

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

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.

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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

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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Treatment Virus RNA Therapies 148

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.

Therapies 130

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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

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

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. Before giving the treatment, researchers monitored the mice’s brain activity for 15 days.

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Therapies Virus Regulations Clinical Trials 265

Drug Discovery World

AUGUST 21, 2024

Dr Andrea O’Hara, Senior Product Manager at Azenta Life Sciences asks why integration site analysis (ISA) is critical for the safety and efficacy of viral vectors for gene therapy. Cell and gene therapies have revolutionised how clinicians treat a variety of diseases including cancer, blindness, and metabolic disorders.

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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 290

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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Disease Therapies Government Treatment 148

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 265

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

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

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.

Therapies 148

Therapies Disease Medical Schools Trials 148

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.

Therapies 130

Therapies Engineering Treatment Regulations 130

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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Disease Therapies International Drug Development 148

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 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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Therapies Clinical Trials Trials Government 279

Drug Discovery World

AUGUST 6, 2024

Circular RNAs (circRNAs) have “remarkable potential” to improve cancer treatment and patient outcomes within the next five to 10 years, according to a new study by Flinders University in Australia. We’ve also discovered that high levels of circRNAs in certain people can cause mutations in their DNA which results in leukaemia.”

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Therapies RNA Immune Response DNA 148

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 148

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 148

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

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 Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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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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Therapies Small Molecule Production Treatment 246

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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Trials Therapies DNA Small Molecule 148

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. The post Cell and gene therapy: Global Innovation and Opportunity appeared first on Drug Discovery World (DDW). 2024 is potentially shaping up to break this record.

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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

Therapies 278

Therapies Clinical Trials Trials International 278

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

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.

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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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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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