Drug Discovery World

FEBRUARY 2, 2023

The approval of elacestrant is welcomed as it offers a novel option for patients with ER+, HER2- metastatic breast cancer.

FDA Approval 52

FDA Approval Trials Drugs Medicine 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

XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

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Drugs FDA Approval Marketing Development 246

Drug Discovery World

APRIL 4, 2023

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

Drug Discovery World

JULY 7, 2023

In other headline news, the FDA approved the first cellular therapy for type 1 diabetes which could allow some patients to become insulin-independent, and a new study linked severe Covid-19 outcomes with high levels of antibiotics use.

Drugs 52

Drugs FDA Approval Insulin Gene 52

Pharmaceutical Technology

FEBRUARY 15, 2023

These animals have been used as a key tool in functional genomics to generate models for human diseases and to validate new drugs. The Regeneron Genetics Center (RGC) is an integrated research initiative that improves patient care by using genomic approaches to expedite drug discovery and development.

Genetics 130

Genetics DNA Antibody Genome 130

XTalks

JULY 19, 2021

Phosphorus, a leading preventative genomics company, has developed the first comprehensive preventative genetic test for consumers. Phosphorus may be a familiar name as it was one of the first to develop an FDA-authorized at-home test for COVID-19 ; it was also the first test to have received an asymptomatic, direct-to-consumer FDA approval.

Genetics 98

Genetics Genome Genomics Gene 98

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. Despite the concerns, the FDA maintains that the overall benefits of the therapies outweigh the potential risks for their approved uses​​​​.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

pharmaphorum

SEPTEMBER 12, 2022

This reach allows us to support 82% of the novel drugs and therapeutic products approved by the US FDA, including 63% of those specific to oncology,” Reddy states. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

Drug Discovery World

SEPTEMBER 14, 2023

In May 2023, Pfizer announced the FDA approval Abrysvo, the company’s bivalent respiratory syncytial virus (RSV) prefusion F (RSVpreF) vaccine, for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years and older. Read about more recent corporate appointments here.

Drugs 59

Drugs Vaccination Vaccine Medicine 59

pharmaphorum

OCTOBER 27, 2022

It can be caused by different types of mutation, including within the BRCA gene and other forms of altered gene expression. HRD impairs cells’ ability to repair structural DNA breaks, leading to additional changes in the DNA of the tumour (known as genomic instability), and how that tumour will respond to certain treatments.

Trials 92

Trials DNA Genome Genomics 92

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. The future of cell and gene therapies.

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Gene Therapy Gene Marketing Manufacturing 130

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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Research Gene Gene Therapy Genome 264

The Pharma Data

AUGUST 27, 2020

FoundationOne Liquid CDx analyses more than 300 cancer-related genes and multiple genomic signatures to help inform treatment decisions for all solid tumour cancers. FoundationOne Liquid CDx adds to Foundation Medicine’s genomic testing portfolio supporting Roche’s mission to deliver truly personalised healthcare.

FDA Approval 52

FDA Approval Genome Genomics Gene 52

XTalks

NOVEMBER 7, 2022

These tumor-derived entities are used to derive genomic and proteomic data. Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. Regulatory Approvals for CTC-Based Assays in Oncology. The Utility of cfDNA for Cancer Management and Predicting Outcomes.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Pharmaceutical Technology

DECEMBER 22, 2022

Finally there was also the completion of a deal for Biohaven Pharmaceuticals, which adds a calcitonin gene-related peptide portfolio, including NURTEC® ODT, to the company’s portfolio. Sanofi wins the Product Launches award this year following the further development of its Dupixent (dupilumab) product and a string of approvals.

Insulin 130

Insulin Production Development Genetics 130

XTalks

MARCH 28, 2024

Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, gene editing can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

Gene Therapy 110

Gene Therapy Gene Trials FDA Approval 110

Delveinsight

DECEMBER 15, 2020

Sold under the name Jakafi, it is FDA-approved for the treatment of polycythemia vera (PV) in adults. In a historic first, the US FDA issued an Emergency Use Authorization (EUA) to the COVID-19 mRNA, BNT162b2, vaccine candidate for COVID-19 of Pfizer-BioNTech. Welcome the new entrant in Gene therapy market: InnoSkel.

Vaccination 54

Vaccination Vaccine Gene Therapy Gene 54

pharmaphorum

MARCH 15, 2022

Gene sequencing specialist Illumina has started rolling out a new test that looks for a battery of tumour-associated genes from a single tissue sample, and could be used to help guide treatment for a broad range of cancers. “Comprehensive genomic profiling has proven to be this platform.”

Gene 52

Gene Genome Genomics Gene Sequencing 52

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

Production 52

Production Gene Protein Vaccination 52

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic. CAR T cells are T cells that have been trained to attack tumours.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

XTalks

AUGUST 12, 2020

Related: FDA Approves Rukobia for HIV Patients with Limited Treatment Options. The results of persistent, underlying HIV infection include heightened immune activation and inflammation (leading to a number of diseases), ongoing damage to multiple organ systems and reduced life expectancy.

RNA 52

RNA Protein DNA In-Vivo 52

pharmaphorum

SEPTEMBER 13, 2022

Just over a decade after it was developed by biochemist Nicholas Lyndon, Imatinib received US Food and Drug Administration (FDA) approval in 2001. 2012 – The 100,000 Genomics Project begins. Unlocking the secrets of the human genome has intrigued investigators for centuries. 2002 – Emergence of CAR-T therapy.

Genome Project 98

Genome Project Genome Genomics DNA 98

XTalks

SEPTEMBER 12, 2023

Biomedical Research and Technologies: Get access to the most recent biomedical research news from both industry and academia, including preclinical studies and technological breakthroughs in genomics, single cell analysis, proteomics, metabolomics and much more.

Life Science 105

Life Science Branding Clinical Trials Clinical Trials 105

Drug Discovery World

JANUARY 17, 2024

ctDNA carries fragments of genomic information, whereas CTCs carry whole sequences, facilitating not only genomic, but transcriptomic and proteomic analysis. 9 Next generation sequencing (NGS) is fuelling a revolution in liquid biopsy analysis, providing genomic and transcriptomic characterisation for personalised therapy selection.

Medicine 59

Medicine DNA Genome Genomics 59

Camargo

DECEMBER 10, 2021

Biomarin’s stock shot up after the drug was first approved in the EU this past August, and on the day the news broke of the FDA approval, the stock improved 10.3%. Subsequent revisions of the document reflect the experience gained as gene therapy products of this type have been approved.

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Development Genetics Production Clinical Trials 176

The Pharma Data

OCTOBER 14, 2020

By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs, NantKwest’s goal is to transform medicine by bringing novel NK cell-based therapies to routine clinical care. Cancer Gene Ther. iv Osada T, et al.

Antibody 52

Antibody Vaccination Vaccine Trials 52

Drug Discovery World

AUGUST 1, 2023

Antisense Oligonucleotides (ASOs) ASOs are a type of nucleotide-based therapeutic that are short, synthetic, chemically modified chains of nucleotides that have the potential to target any gene product of interest 2. AstraZeneca’s non-alcoholic steatohepatitis (or ‘NASH’) study is a prime example of the value of ASOs 2.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

XTalks

MARCH 25, 2021

Biomedical Research and Technologies : Presenting the latest biomedical research from both industry and academia, including preclinical studies as well as technological innovations in genomics, single cell analysis, proteomics, metabolomics and much more.

Life Science 105

Life Science HR Clinical Trials Clinical Trials 105

The Pharma Data

JANUARY 4, 2021

Food and Drug Administration (FDA) , in combination with Tecentriq ® (atezolizumab) for the first-line treatment of people with metastatic non-small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression with no EGFR or ALK genomic tumor aberrations. Their tumor does not have an abnormal “EGFR” or “ALK” gene.

Immune Response 52

Immune Response Medicine Protein Gene 52

Drug Discovery World

APRIL 17, 2023

DDW’s Megan Thomas rounds up highlights from Monday 16 April at AACR 2023, from promising trial results for colorectal cancer patients to updates on Biden’s Cancer Moonshot initiative. AACR-Cancer Research Institute Lloyd J. Old Award in Cancer Immunology Another award highlight was the AACR-Cancer Research Institute Lloyd J.

Research 52

Research Antibody Bioavailability Genetics 52

Delveinsight

JULY 20, 2021

The AllplexTM SARS-CoV-2/FluA/FluB/RSV Assay can simultaneously detect and differentiate a total of five viruses including Flu A, Flu B, RSV A/B, and three target genes of COVID-19 (N gene, S gene, and RdRP gene) in a single test. Laboratory Director for Wren.

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FDA Approval Marketing Trials Gene 96