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

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 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

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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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.

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BioPharma Drive: Drug Pricing

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

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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. MT: What’s next for STORM’s research?

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. Dr Samulski has advanced therapeutics into human clinical trials for haemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure. He holds more than 200 patents related to AAV technology.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. Dr Samulski has advanced therapeutics into human clinical trials for haemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure. He holds more than 200 patents related to AAV technology.

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Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . One trial participant was Vincenzo Mascoli, aged 22. This treatment made a huge difference in quality of life for Vincenzo and other trial participants.” .

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Drug Discovery World

AUGUST 2, 2022

Biotechnology company Calidi Biotherapeutics has announced that a Phase I US clinical trial will use the company’s licensed oncolytic virotherapy platform NeuroNova to deliver an oncolytic adenovirus selectively to tumor sites in patients with recurrent high-grade glioma. .

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Drug Discovery World

AUGUST 8, 2023

Astellas Pharma will invest $50 million into Poseida Therapeutics to secure the rights to the company’s cancer CAR-T cell therapy for solid tumours, which is currently in Phase I trials. ” Astellas’ portfolio includes oncolytic viruses, bispecific immune cell engagers, small molecules, and cell therapy platforms.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JANUARY 25, 2023

The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge Biologic’s adeno-associated virus (AAV) drug candidate for treating patients with Krabbe disease. The post EMA prioritises AAV gene therapy for Krabbe disease appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 9, 2024

The top stories: Retinoblastoma therapy gets FDA rare paediatric drug designation Theriva Biologics’ VCN-01 for retinoblastoma has been granted rare paediatric drug designation (RPDD) by the US Food and Drug Administration (FDA). The post This week in drug discovery (5-9 August) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JUNE 28, 2024

This week has seen some exciting advances in neuroscience drug discovery, including Phase II clinical trials for two potentially game-changing treatments, a synapse-regenerating drug for Alzheimer’s and a gene therapy for Parkinson’s, as well as the launch of DDW’s latest In Focus report.

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Drug Discovery World

JULY 15, 2024

The Cell and Gene Therapy Catapult (CGT Catapult) has established a cross-industry consortium to improve the understanding and process optimisation of adeno-associated virus (AAV) formulation. AAVs are the most popular gene delivery system currently used in gene therapy clinical trials.

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Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields.

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Drug Discovery World

APRIL 3, 2023

The findings indicate that a drug compound, R805/CX-011, may modulate an important cell receptor in the body’s immune system, GP130, that signals when antibodies should attack a virus or infection. The post Potentially ‘regenerative’ osteoarthritis drug moves to clinical trial appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Drug Discovery World

JULY 26, 2022

BM: Calidi Therapeutics’ current pipeline consists of oncolytic virus-based immunotherapies potentiated by allogeneic stem cells and designed to treat patients for whom few, if any, effective treatment options exist. Both therapies also had excellent safety profiles. RA : Could you give us an idea of your current pipeline?

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Drug Discovery World

OCTOBER 27, 2023

My news highlights this week feature a variety of treatment approaches, from cell therapy and antibody-drug conjugate to autotaxin inhibitor and arenaviral vaccine, all entering early in-human clinical trials to treat a range of diseases.

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Drug Discovery World

SEPTEMBER 22, 2023

As we approach another autumn/winter season in the northern hemisphere, Covid-19 vaccines are once again making the headlines, but in the last week we have also reported on vaccine trials for a number of other conditions, including Nipah virus, rabies and lung cancer.

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Drug Discovery World

MARCH 8, 2024

The top stories: Antibody therapy shows anti-tumour activity in advanced cancer 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.

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. For example, a site conducting only protocols involving mRNA may be able to leverage a generic SOP covering any mRNA protocol at that site.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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Drug Discovery World

JUNE 6, 2024

The combination therapy was also shown to reduce the risk of distant metastasis or death by 62% compared to Keytruda alone in these patients. The global Phase III clinical trial is being led by University College London Hospitals NHS Foundation Trust in the UK and will include around 1,100 people.

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Drug Discovery World

OCTOBER 27, 2023

HB-400, a novel arenaviral therapeutic vaccine developed by HOOKIPA Pharma and Gilead Sciences, has been shown to generate robust T cell responses specific to hepatitis B virus with high antibody levels in a preclinical setting. A Phase I clinical trial to evaluate the safety and tolerability of HB‑400 in humans is ongoing.

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Drug Discovery World

JUNE 10, 2024

The US Food and Drug Administration (FDA) has approved mRESVIA (mRNA-1345), an mRNA respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection. This approval is also the first time an mRNA vaccine has been approved for a disease other than Covid-19.”

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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Drug Discovery World

AUGUST 1, 2024

DDW Editor Reece Armstrong speaks to Dr Steven Powell , co-founder of Glen Clova Scientific (GCS), about the company’s approach of using virus-like particles (VLPs) to treat conditions such as eczema and asthma, and what the current market looks like for this therapy area. Is there a large unmet need?

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Vial

DECEMBER 11, 2023

Hepatitis C virus (HCV) infection is a global health problem with complications that place a significant economic burden on national health systems. Other inhibitors, currently in different stages of development (including clinical trials ), mainly target viral non-structural proteins or the internal ribosome entry site (IRES).

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Drug Discovery World

OCTOBER 20, 2023

GenomeFrontier partners with BioCina on CAR-T therapy CDMO BioCina and GenomeFrontier Therapeutics will partner on the development of virus-free CAR-T products for cancer treatment. BioCina will provide process development and GMP manufacturing for Minicircle DNA and Plasmid DNA.

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Drug Discovery World

JANUARY 9, 2024

They include therapeutics for conditions including breast cancer, haemophilia A, sickle cell disease, Crohn’s disease, ulcerative colitis, respiratory syncytial virus (RSV) and multiple myeloma, among others. The therapy has shown positive results in Phase III trials in both breast and lung cancer.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Drug Discovery World

JUNE 16, 2023

The top stories: FDA approves Phase III cerebral Adrenoleukodystrophy trial THE FDA has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

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