Drug Discovery World

AUGUST 5, 2024

Adaptimmune Therapeutics’ Tecelra (afamitresgene autoleucel) has become the first engineered cell therapy for a solid tumour cancer approved in the US. It is also the first new therapy option in more than a decade for synovial sarcoma, which is a rare, soft tissue cancer that most commonly impacts young adults.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . There are currently no approved disease modifying therapies for Huntington’s Disease. In 2019, BV-101 was granted orphan drug designation in the European Union. .

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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

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

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

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Drug Discovery World

AUGUST 30, 2024

Dr Nicholas Siciliano, Co-Founder of Vittoria Biotherapeutics, shares insight on the VIPER-101 trial targeting T-cell lymphoma, the company’s aim to ensure progression free survival and opportunities in this sector. MT: I understand that your lead programme is moving into the clinic, with POC human data expected in 2024.

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Drug Discovery World

APRIL 12, 2024

The company plans to initiate a Phase I study of the investigational allogeneic off-the-shelf Tr1 Treg therapy for this indication in the second quarter of 2024. ” Diana Spencer, Senior Digital Content Editor, DDW The post FDA green light for trial of allogeneic regulatory T cell therapy appeared first on Drug Discovery World (DDW).

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Science Daily: Pharmacology News

SEPTEMBER 6, 2024

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

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Drug Discovery World

SEPTEMBER 4, 2024

The first patient has been treated in a Phase II trial of EVO756 in adults with chronic inducible urticaria (CIndU). “As we continue to execute on our clinical development plans for EVO756, we are commencing this Phase II trial in 15 study sites across the United States.

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Drug Discovery World

NOVEMBER 20, 2023

“This study aims to build upon the promising results from our previous Phase II trial of CVN424 as an adjunctive therapy for later-stage Parkinson’s disease. CVN424 is a first-in-class non-dopamine therapy that selectively modulates GPR6, an orphan G-protein coupled receptor.

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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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Therapies Trials Treatment Disease 307

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

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.

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Therapies Clinical Trials Trials International 278

Drug Discovery World

MARCH 4, 2024

In an early phase clinical trial, a combination of antibody-based medications targeting the immune system generated promising safety data and anti-tumour activity in individuals with advanced cancer. Both medications tested in the trial support immune responses against tumour cells. Various dosing schedules of CS1002 (0.3,

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Therapies Immune Response Clinical Trials Trials 290

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). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Therapies Clinical Trials Trials Treatment 278

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 early-stage clinical trial was the result of a collaboration between researchers at University of Colorado Anschutz Campus, the University of Cambridge and the University of Milano-Bicocca. “We

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Therapies Clinical Trials Trials Treatment 265

Drug Discovery World

JUNE 15, 2023

A Phase I clinical trial testing a CAR-T cell therapy treatment in children with acute myeloid leukaemia (AML) has been paused due to a patient’s death. The PLAT-08 clinical trial is a Phase I study of 2seventy bio’s cell therapy SC-DARIC33 in patients with AML.

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Clinical Trials Therapies Trials FDA 130

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. It is currently also being evaluated in Phase I/II and Phase II trials across two broad areas of autoimmune disease: rheumatology and neurology. “On

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Therapies Disease Hospitals Treatment 246

BioPharma Drive: Drug Pricing

JUNE 12, 2024

The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.

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Therapies Trials 117

Drug Discovery World

AUGUST 7, 2024

The US Food and Drug Administration (FDA) has cleared GigaGen’s application to initiate a Phase I trial of its polyclonal drug for the treatment of hepatitis B virus (HBV) infection, GIGA-2339. With over 1,000 different HBV-targeted antibodies in the mixture, GIGA-2339 is unlike any therapy currently in development.

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Drug Trials FDA Trials Immune Response 148

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). I look forward to the data that will emerge from the KYSA-8 trial as this trial could drastically change the treatment landscape for SPS.”

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Therapies FDA Trials Treatment 130

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.

FDA Approval 243

FDA Approval Therapies FDA Protein Expression 243

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. The company’s Phase II REGENERATE-PD clinical trial is currently enrolling patients in the US with sites in the European Union and the UK planned to open later this year.”

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Therapies Clinical Trials FDA Clinical Development 148

Drug Discovery World

AUGUST 5, 2024

Most recently, results from the investigator sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in paediatric patients with refractory retinoblastoma were determined to be positive by the study Monitoring Committee.”

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FDA Therapies Drugs Clinical Trials 148

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 researchers plan to conduct a first-in-human clinical trial in the next five years. The team then monitored the mice’s brain activity for another 15 days.

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Therapies Virus Regulations Clinical Trials 265

Drug Discovery World

JUNE 25, 2024

A new study demonstrates that AI-powered digital twin solutions can potentially replace the standard-of-care (SOC) control arm of a clinical trial. We have demonstrated that digital twins offer real potential to replace standard-of-care comparator arms to streamline the implementation of clinical trials and dramatically reduce patient burden.”

Clinical Trials 148

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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

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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Therapies Clinical Trials Trials Production 180

Drug Discovery World

JULY 24, 2024

CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC. This extension to the companies’ collaboration agreement will see Evotec significantly broaden the pipeline with additional ASOs as candidate therapies. They are instrumental in supporting us as we prepare for those clinical trials.”

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Therapies Disease Clinical Trials Hospitals 130

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.

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Therapies Engineering Treatment Regulations 130

Drug Discovery World

AUGUST 9, 2024

The first patient has been dosed in a Phase I clinical trial of AGX101, a novel TM4SF1-directed antibody-drug conjugate being developed for the treatment of solid cancers. We look forward to evaluating AGX101 in this trial.”

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Trials Pharmacokinetics Drugs Clinical Trials 148

Science Daily: Pharmacology News

JUNE 5, 2024

A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.

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Therapies Trials Clinical Trials International 114

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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Therapies Clinical Trials FDA Approval Virus 163

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.

Therapies 148

Therapies Clinical Development Disease FDA 148

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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FDA Approval FDA Therapies Treatment 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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Therapies Engineering Treatment Pharma Companies 130

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