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

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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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).

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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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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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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 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

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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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

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

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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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

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.

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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

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

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

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

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

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

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.

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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

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 2.7

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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. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

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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 experiment was based on previous success with CAR-T therapy. The treatment team in Erlangen has now published the results of this successful treatment in the medical journal The Lancet.

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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

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

MAY 8, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post How can long-read nanopore sequencing support gene therapy delivery? appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 23, 2024

Given the increased awareness of concussion and the associated long-term sequela from concussive injury it is imperative that we accelerate drug development efforts to find an effective therapy. Tremendous strides have been made with other diseases, yet there are still no FDA-approved drug therapies for TBI.

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Drug Discovery World

JULY 11, 2024

The post Infographic: Overcome your cell and gene therapy (GMP) challenges appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 30, 2024

The eReport includes: In-depth analysis of regional markets An overview of the likely challenges and opportunities The benefits of allogeneic CAR-T cell therapies The latest advances and where could CGTs take us in the future The post Cell and gene therapy: Global Innovation and Opportunity appeared first on Drug Discovery World (DDW).

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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

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. It is imperative for the industry to communicate the precise composition and attributes of these therapies to regulatory bodies.

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Drug Discovery World

AUGUST 30, 2022

The Phacilitate Advanced Therapies Europe 2022 conference will take place from 30 August to 1 September at the Royal Lancaster in London, UK. The conference is the European twin to Phacilitate Advanced Therapies Week, an event held in January in Florida, US. Creating a sustainable cell & gene therapy company.

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JANUARY 31, 2024

Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor. Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs.

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Drug Discovery World

NOVEMBER 27, 2023

This 24-page eBook sponsored by Sartorius explores the latest innovations in cell and gene therapies, where the challenges lie and the possible solutions to overcoming these obstacles.

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Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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Drug Discovery World

AUGUST 4, 2023

Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world. Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world.

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