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

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

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

SEPTEMBER 9, 2024

SYNCAR-001 + STK-009 is a cytokine-inducible, two-component therapy consisting of SYNCAR-001, an autologous CD19-targeting CAR-T which expresses an engineered IL-2 receptor allowing it to selectively receive a signal from STK-009, an engineered IL-2 cytokine.

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Drug Discovery World

JANUARY 5, 2024

CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body. “This very important study will answer whether CAR T-cell therapy offers a new treatment option for patients living with MS. .

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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. The post Gene therapy could reduce seizures in childhood epilepsy appeared first on Drug Discovery World (DDW).

Therapies 264

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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

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

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

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?

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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

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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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

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

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.

Disease 147

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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

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

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

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).

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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

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

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

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

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 289

Therapies Immune Response Clinical Trials Trials 289

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. Perhaps most significantly, Casgevy (or exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, was approved to treat sickle cell disease and became the first gene therapy that uses CRISPR gene editing.

Therapies 147

Therapies FDA Clinical Research Marketing 147

Drug Discovery World

AUGUST 5, 2024

Diana Spencer, Senior Digital Content Editor, DDW The post Retinoblastoma therapy gets FDA rare paediatric drug designation appeared first on Drug Discovery World (DDW).

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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).

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Drug Discovery World

APRIL 19, 2024

Autolomous and BioCentriq have collaborated to digitise and accelerate process development and manufacturing activities for cell therapy. Autolomous’ software platform is designed to materially affect the cell and gene therapy sector.

Therapies 130

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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 therapy is a humanized monoclonal antibody engineered to neutralise the tumour-produced Growth Differentiation Factor-15 (GDF-15), which acts as a key regulator of immune resistance to cancer therapies.

Therapies 130

Therapies Engineering Treatment Regulations 130

Drug Discovery World

JULY 12, 2024

AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy. AskBio is also exploring GDNF therapy beyond Parkinson’s disease and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I trial.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Drug Discovery World

AUGUST 22, 2024

The therapy’s efficacy was evaluated in the Phase III MARIPOSA trial, which included 1074 patients with exon 19 deletion or exon 21 L858R substitution mutation-positive locally advanced or metastatic NSCLC and no prior systemic therapy for advanced disease.

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Drug Discovery World

AUGUST 8, 2024

They are not curable with current therapies and without treatment they continue to grow and infiltrate normal brain tissue. We are proud to deliver this first-of-its-kind therapy to patients in need, and we remain committed to bringing innovative targeted therapies to people with cancer.”

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Drug Discovery World

JUNE 11, 2024

The European Medicines Agency has approved a Type II variation to the Summary of Product Characteristics (SmPC) for Kite’s chimeric antigen receptor (CAR)-T cell therapies, Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel).

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Drug Discovery World

JUNE 26, 2024

The gene therapy EG110A is a non-replicating HSV-1 vector that has been designed to selectively silence the signals of key bladder sensory neurons responsible for the bladder muscle overactivity, whilst preserving motor neuron and retaining normal bladder function. “NDO

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