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

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Delveinsight

FEBRUARY 18, 2021

Bluebird suspends gene therapy trials after two sickle cell patients develop cancer. Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

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Roots Analysis

FEBRUARY 27, 2024

Over the last few decades, various viral vector manufacturing have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. BacTRL Gene Therapy Platform.

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Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

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XTalks

DECEMBER 4, 2023

In a statement outlining its investigation, the FDA said it received reports of the T-cell malignancies from clinical trials and post-marketing safety monitoring. The currently approved therapies target CD19 and BCMA, which are expressed in cancers like B-cell lymphoma and multiple myeloma.

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XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. The Phase I trial for HB-500 is scheduled to start in the first half of 2024.

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Delveinsight

DECEMBER 3, 2020

Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences. There are presently no medical treatments, which can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this arena.

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STAT News

NOVEMBER 4, 2022

Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

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NY Times

FEBRUARY 22, 2021

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

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pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. First of all it will be used for ultra-rare monogenic disease.

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XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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XTalks

MARCH 13, 2024

XTALKS WEBINAR: Addressing Ophthalmology Clinical Trial Design and Effective Data Monitoring Committee Strategies On-Demand: Thursday, February 8, 2024 Register for this free webinar gain insights into statistical data strategies for ophthalmology clinical trials and learn best practices for establishing and reporting ophthalmic data.

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Delveinsight

DECEMBER 17, 2020

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). Instead of directly targeting existing mitochondria, the team went after mitochondrial DNA transcription that is vital to the formation of new mitochondria and the production of energy. million in financing.

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XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.

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pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. .

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Pharmaceutical Technology

FEBRUARY 20, 2023

If clinical trial participants are representative of the wider disease population, they can help sponsors assess the effectiveness of new and existing therapeutics. of the clinical trial population globally. participation of white subjects. The technology platforms are almost the same.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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Pharmaceutical Technology

FEBRUARY 20, 2023

The two companies, along with Cerba Research’s specialty virology subsidiary Viroclinics-DDL, have had a long track record in collaborative projects since 2012, from specialty assay transfers to full clinical trial management and logistics support services. Particularly, Teddy Lab Wuxi set up in 2020 with the great help of Wuxi Government.

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Cloudbyz

JUNE 26, 2023

Clinical Trial Design and Optimization: Generative AI can aid in the design and optimization of clinical trials. By simulating patient populations, generating synthetic trial data, and optimizing trial protocols, generative models can enhance the efficiency and effectiveness of clinical trials.

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Pharmaceutical Technology

MAY 9, 2023

The clinical trials landscape is evolving more rapidly than ever before. Meanwhile, decentralisation and the Big Data revolution are transforming the way researchers run clinical trials, and previously untapped geographies are emerging as new hubs for future research. Clinical trials management is no exception.

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Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. and France to accelerate the development of genomic therapies. About Whitelab Genomics.

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Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We Novartis today announced that the U.S.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. From a technical standpoint, such diseases should be easier to treat with current gene therapy technology.

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The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient’s cells.

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Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Early phase clinical trials for gene editing therapies.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed.

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Drug Discovery World

AUGUST 2, 2022

CureHeart is using gene therapy technologies to edit or silence the faulty genes that cause inherited heart muscle diseases. . Also known as genetic cardiomyopathies, they often go undiagnosed and are a major cause of heart transplants due to how current treatments do not prevent the condition from progressing. .

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Drug Discovery World

AUGUST 10, 2022

Exactly three decades later, the first CAR T cell therapy hit the market. And now, with more than 600 CAR T trials recruiting or ongoing, these cells represent a majority of the cellular immunotherapies in development today. They’ve also induced lymphomas in humans when delivered directly in the form of gene therapy 17.

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The Pharma Data

MARCH 20, 2023

2 “I have had the privilege of observing some of the children included in the LTFU studies since they started their Zolgensma clinical trial journey, and the fact that we’re seeing them maintain and, in some cases, gain motor milestones when they are nearly eight years old is truly transformational,” said Dr. Jerry R.

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Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. These methods offer safer CAR T-based therapies, laying the foundation for clinical trials and the rapid development of a new generation of CAR T cells for both autologous and allogeneic use.

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The Pharma Data

DECEMBER 27, 2020

Even with the holidays among us, there were a number of clinical trial announcements. Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Here’s a look. COVID-19-Related. Non-COVID-19-Related.

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Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. The first in-person iPSC-derived regenerative therapy trial took place at Kyoto University, Japan.

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