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 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

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

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

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

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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

JANUARY 5, 2024

Kyverna Therapeutics has revealed plans to progress to Phase II trials of KYV-101 for multiple sclerosis (MS) following a green light from the US Food and Drug Administration (FDA). CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body.

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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

SEPTEMBER 15, 2023

Advanced Therapies Europe took place from 9-12 September 2023 in Portugal. DDW’s Megan Thomas asked attendees: What is most important to scale from discovery to commercialisation of cell and gene therapies (CGTs)? Joel Eichmann, Co-Founder, Green Elephant Biotech It always comes down to time-to-market and risk minimisation.

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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.

Therapies 265

Therapies Virus Regulations Clinical Trials 265

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. Diana Spencer, Senior Digital Content Editor, DDW The post First use of CAR-T therapy in patient with stiff-person syndrome appeared first on Drug Discovery World (DDW).

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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. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?

Therapies 307

Therapies Trials Treatment Disease 307

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

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

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. 2024 is potentially shaping up to break this record.

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Therapies FDA Clinical Research Marketing 148

Drug Discovery World

AUGUST 7, 2024

.” Shinobi’s immune evasive iPS-T cell technology, built upon a decade of research by co-founders Shin Kaneko at Kyoto University and Tobias Deuse at University of California, San Francisco, enables a new class of off-the-shelf cell therapies.

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Drug Discovery World

SEPTEMBER 20, 2023

A new cooperative network designed to foster collaboration and boost the sharing of knowledge and expertise for the UK’s advanced therapies sector has been launched.

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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 post Stem cell therapy shows promise in advanced MS appeared first on Drug Discovery World (DDW). But this is an encouraging step towards a new way of treating some people with MS.”

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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). The therapy has already shown great promise in preclinical studies, and we now look forward to taking this important next step in its clinical development.”

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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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Therapies Research Clinical Trials Science 130

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 19, 2024

If CV6-168 fulfils its’ promise, then this will give us a new therapeutic option that is easy to take alongside existing standard anti-cancer drugs, and with minimal additional side-effects.” In preclinical studies, the drug has demonstrated robust anti-tumour activity in cell line and animal models with no added toxicity.

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Trials Therapies DNA Small Molecule 148

Drug Discovery World

JULY 24, 2024

SynaptixBio and Evotec have announced they are extending their partnership to find further candidate drugs to treat H-ABC, the most severe form of TUBB4A leukodystrophy. CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC.

Therapies 130

Therapies Disease Clinical Trials Hospitals 130

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 6, 2024

New data has revealed a robust microdystrophin expression from a pivotal dose of gene therapy RGX-202 for Duchenne muscular dystrophy (Duchenne). It is the only gene therapy approved or in development for Duchenne that encodes for the C-Terminal (CT) domain to produce a microdystrophin that is closer to naturally occurring dystrophin.

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Drug Discovery World

MARCH 26, 2024

DDW’s Megan Thomas caught up with Oliver Rausch, Chief Scientific Officer at Storm Therapeutics, following the ESMO Targeted Anticancer Therapies Congress 2024, a global meeting focusing on promising new anticancer targets and agents, focusing on those in early phase clinical development.

Therapies 163

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Drug Discovery World

JULY 12, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy.

Therapies 148

Therapies Clinical Trials FDA Clinical Development 148

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). It is estimated that this includes around 2,000 patients in the UK. “I

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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

OCTOBER 9, 2023

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. The post Non-viral gene therapy shows early efficacy in lung cancer appeared first on Drug Discovery World (DDW).

Therapies 278

Therapies Clinical Trials Trials International 278

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 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.

FDA Approval 148

FDA Approval FDA Therapies Treatment 148

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,

Therapies 290

Therapies Immune Response Clinical Trials Trials 290

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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Therapies Disease Medical Schools Trials 148

Drug Discovery World

JUNE 26, 2024

Biotechnology company EG 427 has revealed plans to start a Phase Ib/IIa clinical study in neurogenic detrusor overactivity (NDO), following Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA).

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Therapies Clinical Development Disease FDA 148

Drug Discovery World

MARCH 6, 2024

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination.

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Disease FDA Therapies Drugs 130

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). KYV-101 is an autologous, fully human CD19 CAR-T cell product candidate for use in B cell-driven autoimmune diseases.

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