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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Gene Therapy Gene Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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Gene Therapy FDA Approval Gene In-Vivo 98

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. Zinc fingers.

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Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. He added: “We have seen no significant gene misregulation attributable to the insertion event.

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Gene Therapy Gene Trials Gene Expression 59

pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

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Gene Therapy Gene DNA Licensing 106

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

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Gene Therapy Gene Genetic Disease DNA 94

Pharmaceutical Technology

MARCH 28, 2023

On March 27, researchers presented new interim results from the Phase III RUBY clinical trial that could indicate a new standard of care for endometrial cancer treatment. GSK’s trial evaluated the programmed death receptor-1 (PD-1)-blocking antibody Jemperli (dostarlimab). GlobalData is the parent company of Pharmaceutical Technology.

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DNA Clinical Trials Clinical Trials Trials 130

Scienmag

JANUARY 20, 2021

Study supports launch of Phase I clinical trial to test a designer DNA agent — an antisense oligonucleotide that targets a gene called IRF4 — in patients with multiple myeloma Many patients with multiple myeloma, a type of blood cancer, eventually develop resistance to one treatment after another.

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DNA Clinical Trials Clinical Trials Gene 56

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Gene Therapy Gene Genome Genomics 105

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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Clinical Trials Clinical Trials Vaccination Vaccine 75

Drug Discovery World

OCTOBER 11, 2022

A team of researchers at Northwestern University in the US has devised a new platform for gene editing that could inform the future application of CRISPR-based therapeutics. They created a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. High gene editing efficiency.

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Gene Editing Gene DNA Protein 52

Pharmaceutical Technology

JUNE 21, 2023

The combination therapy is indicated to treat homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) in adult patients. Talzenna is an oral inhibitor of poly ADP-ribose polymerase (PARP) that plays a role in repairing DNA damage.

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FDA Approval Gene DNA Medicine 130

Pharmaceutical Technology

APRIL 12, 2023

FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients. Exposure to environmental factors can cause gene fusion which leads to upregulation of the ROS-1 enzyme.

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Genome Genomics In-Vitro Gene 130

XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. Boorjian, MD, Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin, in the company’s press release.

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Gene Therapy FDA Approval Gene Clinical Trials 52

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). Cortexyme tripped up by FDA hold over liver toxicity.

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Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

AUGUST 2, 2023

New findings suggest PARP inhibitor drugs, used to treat cancer patients who have an altered BRCA1 or BRCA2 gene, may also be effective for patients which have changes in the SF3B1 gene. The researchers discovered that PARP inhibitor drugs reduced the ability of cancer cells with an altered SF3B1 gene to survive.

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Gene DNA Clinical Trials Clinical Trials 52

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. This means site personnel do not have to review and sign a new SOP each time they submit a new mRNA protocol.

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Genetic Engineering Clinical Trials Clinical Trials Engineer 52

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? Skills gap Bruce Levine, International Society for Cell & Gene Therapy (ISCT) Immediate Past President and Chair, Strategic Advisory Council, says a critical challenge that has become apparent for the CGT sector is the need for workforce development and training resources.

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Gene Therapy Gene Manufacturing Marketing 52

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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Manufacturing Gene Therapy Gene DNA 52

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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Gene Therapy Gene Manufacturing Medicine 52

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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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

DECEMBER 8, 2023

The news highlights this week are mostly focused around neuroscience, with new guiding principles and a drug repurposing fund launched for motor neuron disease research, a new drug target found for early-onset dementia, and a possibly game-changing schizophrenia drug entering human trials.

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Drugs DNA Clinical Trials Clinical Trials 52

XTalks

NOVEMBER 7, 2022

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. Liquid Biopsy Use in Oncology Clinical Trials.

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Clinical Trials Clinical Trials Trials FDA Approval 98

pharmaphorum

NOVEMBER 22, 2021

This forum will specifically focus on targeting mitochondrial dysfunction to develop therapies for chronic diseases caused by mitochondrial DNA mutation/nuclear DNA mutations, muscle dystrophy diseases/muscle loss, metabolic disorders, neurodegenerative disease, and conditions linked to oxidative damage.

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Development Drugs DNA Gene Therapy 98

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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Gene Therapy Marketing Gene DNA 52

Pharmaceutical Technology

DECEMBER 14, 2022

Bacteria naturally develop resistance to antibiotics over time, through genetic mutation, horizontal gene transfer between bacteria, or because of selective pressure. In the same way that viruses attack human cells and replicate using machinery in the human cell, phages insert their DNA into a bacterial cell through transduction.

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Bacteria DNA Clinical Trials Clinical Trials 130

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

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Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). The drug candidate is administered through the oral route in the form of capsule and tablet. It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Drug Discovery World

AUGUST 5, 2022

Evonetix is reimaging biology by developing a different approach to gene synthesis. I began exploring array-based technology for highly parallel DNA synthesis and whether this could be achieved using multiple heater/cooler synthesis sites to overcome some of the challenges faced by traditional array-based DNA synthesis platforms.

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DNA Engineer Gene Sequencing Gene 52

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. This well-read article featured in the DDW Winter 22/23 issue’s Cell & Gene Therapy Guide.

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Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

OCTOBER 25, 2022

Scientists have shown how stomach cancers can develop resistance to a new class of drugs called ATR inhibitors by switching off the activity of two key genes – raising the possibility of outsmarting cancer by predicting drug resistance in advance. . CRISPR used to identify genes involved. It was funded by Cancer Research UK. .

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Development Drugs Gene Editing Gene 52

Drug Discovery World

OCTOBER 10, 2023

Read more: Preclinical study proves antidepressant effects of drug Psychedelic-based drug options Later the same month, Cybin announced it had completed dosing in Cohort 5 of a Phase II trial evaluating CYB003, an investigational proprietary deuterated analog of psilocybin for the potential treatment of major depressive disorder (MDD).

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Drugs Gene Expression Gene Immune Response 59

The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA (mtDNA). FRIDAY, Dec. Congress has barred the U.S.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Drug Discovery World

JUNE 7, 2023

Hosted by DDW and sponsored by DNA Script, join us for this free-to-attend roundtable to learn about the biggest opportunities for the future of drug discovery and development. What can we expect from the cell & gene therapy market? What changes can we expect from clinical trials?

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Development Gene Therapy Drugs DNA 52

Drug Discovery World

AUGUST 16, 2022

Developed by AstraZeneca and Merck & Co (MSD) under the brand name Lynparza, the treatment is a precision medicine which blocks DNA damage response (DDR) in cells or tumours that have a deficiency in homologous recombination repair (HRR), such as those with mutations in BRCA1 and/or BRCA2. . Official comments .

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DNA Gene Medicine Branding 52

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.

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Genetics Gene Hormones RNA 52