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

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

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

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

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

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 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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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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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

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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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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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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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

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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Drug Discovery World

JULY 15, 2024

In June 2023, a patient at Universitätsklinikum Erlangen in Germany became the first child with lupus to receive CAR-T therapy. The treatment was the last resort to slow down systemic lupus erythematosus (SLE), which is a serious autoimmune disease that was attacking the patient’s body and seriously affected her ability to lead a normal life.

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Drug Discovery World

JULY 2, 2024

It is recommended as a monotherapy to treat adult patients with unresectable or metastatic urothelial carcinoma, harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting. months vs 7.8 months vs 2.7

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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

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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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. 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

JUNE 19, 2024

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. Its unique mechanism of action aims to deliver treatment directly to cancer cells, minimising damage to nearby healthy cells 2,5.

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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

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

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

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

JUNE 25, 2024

Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, President and Chief Executive Officer, Sarepta. Diana Spencer, Senior Digital Content Editor, DDW The post FDA expands gene therapy use in Duchenne muscular dystrophy appeared first on Drug Discovery World (DDW).

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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.

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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. million cases of cancer by 2030 1,2.

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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. Matthew Durdy, Chief Executive of the CGT Catapult, said:   “Scotland has a strong life sciences sector.

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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

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

FEBRUARY 28, 2024

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of a Type II variation for Carvykti (ciltacabtagene autoleucel; cilta-cel) for the earlier treatment of relapsed and refractory multiple myeloma (RRMM).

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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thought leadership

MAY 20, 2024

The Challenge: CAGT Treatments Require Time and Lack Availability Persons diagnosed with life threatening diseases which are now treatable with cell and gene therapies (CAGT) often needed to wait for a significant amount of time for treatment due to lack of availability.

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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. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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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 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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