Drug Discovery World

NOVEMBER 17, 2023

Asklepios BioPharmaceutical (AskBio), a gene therapy subsidiary of Bayer, has presented first-in-human data from a Phase I trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF).

Therapies 264

Therapies Treatment Trials Disease 264

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?

Therapies 306

Therapies Trials Treatment Disease 306

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.

Therapies 246

Therapies Small Molecule Production Treatment 246

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.

Therapies 276

Therapies Science Vaccine Production 276

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. Here are a few of many discussions and events to earmark.

Therapies 246

Therapies Hospitals Clinical Trials Clinical Development 246

Drug Discovery World

APRIL 11, 2024

Biopharmaceutical company EpiEndo has completed a Phase IIA clinical trial for its therapy addressing chronic obstructive pulmonary disease (COPD). However, in contrast to antibiotic macrolides, GI adverse events were comparable between the EP395 and placebo-treated participants, and no adverse events related to hearing were reported.

Therapies 130

Therapies Clinical Trials Disease Trials 130

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?

Therapies 147

Therapies Bioinformatics Packaging Molecular Biology 147

Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

Therapies 130

Therapies Disease Clinical Trials Trials 130

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed.

Therapies 130

Therapies RNA Vaccine Small Molecule 130

Drug Discovery World

OCTOBER 3, 2023

Earlier this year, 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.

Therapies 130

Therapies Bioinformatics Packaging Molecular Biology 130

Drug Discovery World

MARCH 19, 2024

The US Food and Drug Administration (FDA) has granted accelerated approval of Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for relapsed or refractory chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL).

FDA Approval 130

FDA Approval Therapies FDA Treatment 130

Drug Discovery World

JUNE 29, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is 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.

Virus 130

Virus Therapies Bioinformatics Packaging 130

Drug Discovery World

JUNE 22, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is 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.

Virus 130

Virus Therapies Bioinformatics Packaging 130

Drug Discovery World

AUGUST 10, 2022

But decades of research led to improvements in cancer therapy, centred around the idea that one’s immune system could aid in treatment. Next, the advent of antibody treatments in 1997 created the first targeted cancer therapy 2. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic.

Therapies 246

Therapies Virus Treatment Engineering 246

SCIENMAG: Medicine & Health

FEBRUARY 26, 2024

The 7th World Conference on Targeting Phage Therapy 2024, two-day event dedicated to advancing the field of phage research and therapy will be hosted at Corinthia Palace Malta on June 20-21.

Therapies 89

Therapies Clinical Trials Research Trials 89

Drug Discovery World

NOVEMBER 24, 2022

DDW’s Diana Spencer asks what will be needed for Europe to compete in the growing global market for advanced therapies. . In terms of ATMPs, other countries are ramping up support for these innovative therapies. China has meanwhile grown its share from 1% to 8%. . Boosting digital capabilities.

Therapies 245

Therapies Clinical Trials Pharmaceuticals Government 245

Drug Discovery World

JANUARY 25, 2023

Cell and gene therapies are transforming the way diseases are treated. As research progresses it is hoped that cell and gene therapies will offer innovation for the future. autologous therapies/solid tumours/ indications outside of oncology?) Register for free now.

Therapies 130

Therapies Drug Development Marketing Disease 130

Drug Discovery World

MAY 24, 2023

Moderna has reported encouraging interim data from its Phase I/II trial of mRNA-3927, an mRNA therapy for rare metabolic disorder propionic acidemia (PA). The data was presented at the 2023 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.

Therapies 130

Therapies Clinical Trials Pharmacokinetics Trials 130

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

FDA Approval 130

FDA Approval Therapies FDA Disease 130

Drug Discovery World

FEBRUARY 9, 2024

DDW recently hosted a webinar ‘Discover how iPSC derived cells can be used to support the development of drug and gene therapies’, supported by DefiniGEN. Chris explained that DefiniGEN is a liver cell company focussed on hepatocytes, particularly supporting the drug development industry in producing liver therapies. Watch on demand.

Therapies 130

Therapies Drugs Drug Development Disease 130

Drug Discovery World

AUGUST 29, 2023

Join DDW for this free event, ‘Discover how iPSC derived cells can be used to support the development of drug and gene therapies’, which is supported by DefiniGEN. The event will take place on 28 September 4PM BST, 5PM CEST, EDT 11AM, PDT 8AM. appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Disease Drugs 130

Drug Discovery World

SEPTEMBER 26, 2022

UMC Utrecht and its partners have received a research grant to unravel how the intestinal microbiota can be exploited to boost cancer immunotherapy and limit therapy side-effects. . These adverse events often present in the intestinal tract as severe inflammation. USA) and MicroViable Therapeutics (Spain). .

Therapies 130

Therapies Research Treatment Disease 130

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.

Clinical Trials 130

Clinical Trials Therapies Trials FDA 130

Drug Discovery World

FEBRUARY 15, 2024

DDW Editor Reece Armstrong speaks to Tracy M Woody , President and Chief Executive Officer of Cessation Therapeutics, about the company’s goal to develop an antibody therapy to prevent fentanyl overdoses. The post Can an antibody therapy prevent fentanyl overdoses? The antibodies stay in the bloodstream and never enter the brain.

Therapies 147

Therapies FDA Approval FDA Government 147

Drug Discovery World

AUGUST 29, 2023

A stem cell derived investigational therapy for treating Parkinson’s disease has shown promise in early clinical trials. The post Stem cell derived Parkinson’s therapy shows promise appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Clinical Trials Trials Disease 130

Drug Discovery World

JANUARY 13, 2023

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment. . This is the largest and longest global Phase III study to date for any gene therapy in haemophilia, with 134 participants. .

Therapies 130

Therapies FDA Licensing Licensing 130

Drug Discovery World

MAY 5, 2023

Efforts to fulfil the promise of precision medicine, including the use of targeted cancer therapies, necessitate that patients be treated based on the specific genetic alterations that drive their individual cancers. But what about the patients whose genomic tests do not identify a cancer driver matched to an existing targeted therapy?

Therapies 130

Therapies RNA Research Pharmaceutical Companies 130

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). . This was a life-changing event for Vincenzo,” said Peter Marinkovich, Associate Professor of Dermatology and Director of Stanford Medicine’s Blistering Disease Clinic.

Therapies 130

Therapies Clinical Trials Trials Virus 130

Drug Discovery World

APRIL 18, 2023

Five patients experienced dose-limiting adverse events (thrombocytopenia and neutropenia), and two such events occurred at the recommended Phase II dose. The post Combo therapy may benefit some paediatric cancer patients appeared first on Drug Discovery World (DDW). cycles of treatment.

Therapies 130

Therapies DNA Trials RNA 130

Drug Discovery World

NOVEMBER 14, 2022

On 4 November 2022, the FDA’s Center for Biologics Evaluation and Research released guidance for the industry for studying multiple versions of a cellular or gene therapy (CGT) product in an early-phase clinical trial in a single disease 1. This guidance finalises the draft guidance of the same title dated September 2021. Key takeaways .

Therapies 130

Therapies FDA Clinical Trials Trials 130

Drug Discovery World

MAY 16, 2023

The UK’s National Institute for Health and Care Excellence (NICE) has recommended additional uses for two CAR-T therapies from Gilead Sciences/Kite, as part of the Cancer Drugs Fund (CDF). The 24-month event-free survival was 40.5% The data supporting its use is based on primary results of the pivotal Phase III ZUMA-7 study.

Therapies 130

Therapies Treatment Science Hospitals 130

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. Record breaking investments in 2021 fuel an industry on fire . billion raised.

Therapies 130

Therapies Clinical Trials Production Disease 130

Drug Discovery World

FEBRUARY 22, 2023

The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW). The treatment was approved by the US Food and Drug Administration in November 2022.

Therapies 130

Therapies Clinical Trials Licensing Licensing 130

Drug Discovery World

NOVEMBER 14, 2023

Of the more than three million people with HeFH in the US and Europe, very few are currently at LDL-C goal, due in part to a care model that requires lifetime therapies. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Trials Clinical Trials Disease 130

Drug Discovery World

APRIL 3, 2024

“Our objective will be to evaluate the tolerance and safety of CM-352 in patients undergoing scheduled total knee replacement surgery (total knee arthroplasty) to prevent possible haemorrhagic complications, which constitute a serious associated adverse event.

Therapies 130

Therapies Laboratories Pharmacokinetics Treatment 130

Chemical Biology and Drug Design

JULY 26, 2023

Abstract Noncoding RNAs (ncRNAs) are engaged in key cell biological and pathological events, and their expression alteration is connected to cancer progression both directly and indirectly. However, there are several limitations, including delivery, uptake, and short half-life, in the application of ncRNAs in cancer treatment.

Engineering 100

Engineering Therapies Cell Biology Drugs 100

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. million price point per dose.

Therapies 130

Therapies Clinical Trials Vaccine Disease 130