XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

FDA Approval 111

FDA Approval Gene Therapy Gene Trials 111

XTalks

FEBRUARY 27, 2023

The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues). A lack of this enzyme results in a toxic build-up of mannose-containing sugars in the cells of the body, including the central nervous system (CNS).

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

XTalks

DECEMBER 1, 2021

The S gene that encodes the characteristic protein harbors a deletion mutation (69-70del), which was also identified in the Alpha variant. Thermo Fisher’s TaqPath COVID-19 assays identify infection by SARS-CoV-2 by detecting the presence of gene targets in the orf1a/b, S (spike) and N (nucleocapsid) regions of the virus.

Gene 105

Gene Genome Genomics Genetics 105

XTalks

OCTOBER 21, 2020

The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. Previous research found fingerprints of positive selection within a gene that encodes the spike proteins.

RNA 98

RNA Protein Genome Genomics 98

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

JUNE 14, 2023

The solution of Vevye does not contain water, anti-microbial preservatives, oils or surfactants. Miebo contains perfluorohexyloctane as its active ingredient, a semifluorinated alkane that forms a protective layer over the tear film, reducing evaporation at the ocular surface. Vevye (cyclosporine ophthalmic solution) 0.1

Gene Silencing 97

Gene Silencing Drug Delivery FDA Approval RNA 97

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This makes Qalsody the first approved treatment to target a genetic cause of ALS.

FDA Approval 89

FDA Approval Gene Genetics Protein 89

XTalks

SEPTEMBER 16, 2022

In fact, one in every 600,000 cells in the lungs of a 50-year-old already contains potentially cancerous mutations. Having a mutation in one copy of either the EGFR and/or KRAS gene isn’t enough to lead to lung cancer. The Double Hit. The new research shows that air pollutants can be that ‘second hit.’

Research 97

Research Gene Scientist Biobanking 97

Drug Discovery World

JANUARY 9, 2024

However, government initiatives to contain healthcare costs, the sustained high cost of capital and global geopolitical disputes are negatively impacting investor appetites for the sector. For patients reluctant to receive novel therapies, such as mAbs or gene therapy, efanesoctocog alfa will likely be an appealing option.

Drugs 69

Drugs Antibody Gene Editing Medicine 69

XTalks

JANUARY 3, 2024

Hereditary transthyretin amyloidosis (hATTR), formerly known as familial amyloid polyneuropathy, is a rare condition caused by mutations in the gene responsible for encoding transthyretin (TTR). This disorder is marked by the extensive deposition of amyloid in extracellular spaces, resulting in dysfunction across various organs and tissues.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

The Pharma Data

JANUARY 7, 2021

Anne Prener has more than 25 years of leadership experience within life sciences companies, and currently serves as Chief Executive Officer of Imbria Inc. a gene therapy company focused on eye diseases. and as venture partner at SV Health Investors. Dr. Prener previously served as CEO of Freeline Ltd. Dr. Prener holds a Ph.D.

Gene Therapy 52

Gene Therapy Life Science Production Clinical Development 52

XTalks

AUGUST 15, 2023

Akeega is a dual-action tablet (DAT) that contains the company’s androgen receptor-targeting hormone therapy Zytiga (abiraterone acetate) and PARP inhibitor (poly [ADP-ribose] polymerase inhibitor) niraparib. Lynparza received FDA approval in May 2020 for mCRPC in patients with homologous recombinational repair ( HRR ) gene mutations.

FDA Approval 52

FDA Approval Gene Hormones Licensing 52

XTalks

JANUARY 9, 2024

New treatments being evaluated in these cancers include Eli Lilly’s Retevmo (selpercatinib) and Blueprint Medicines and Genentech’s Gavreto (pralsetinib), which target mutations in the rearranged during transfection ( RET ) gene. Alternatives to the standard levothyroxine therapy for hypothyroidism are currently being evaluated.

Hormones 105

Hormones Antibody Trials Production 105

The Pharma Data

DECEMBER 28, 2020

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. in Life Sciences and Chemistry from the Weizmann Institute of Science. (Nasdaq: SCPS) today announced the appointment of additional members to its Board of Directors.

Gene Therapy 52

Gene Therapy Life Science Gene Gene Editing 52

XTalks

AUGUST 26, 2020

“When we removed the gene responsible for Rspondin3 from the blood vessel endothelial cells, we observed that macrophages did not decelerate inflammation. The study states, “evolution clearly selected fast healing and containment of injury or infection at the expense of the ability to reform a completely functional tissue.

Bacteria 105

Bacteria Immune Response Protein Medicine 105

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

XTalks

MARCH 10, 2023

It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

FDA Approval 52

FDA Approval Engineer Clinical Trials Clinical Trials 52

XTalks

AUGUST 6, 2020

Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant. CBD is the most abundant non-psychoactive cannabinoid compound found in the cannabis plant, making up about 40 percent of the plant’s extract, which contains over 100 different cannabinoid compounds.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

MARCH 3, 2021

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes. It is characterized by a deficiency in molybdenum cofactor production.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

JUNE 29, 2022

XTALKS WEBINAR: Live Cell Metabolic Analysis — Paving the Way for Metabolic Research and Cell & Gene Therapy. Register for this free webinar to learn how live cell metabolic analysis paves the way not only for metabolic research, but also the manufacturing of significant cell and gene therapy (CGT) products.

FDA Approval 52

FDA Approval Gene Therapy Antibody Gene 52

XTalks

APRIL 13, 2021

synuclein amyloid to a test tube containing the amyloid-forming region of Pmel (known as the repeat, or RPT, domain) and found that the ?-synuclein For example, gene therapy approaches are being investigated to downregulate ?-synuclein -synuclein and Pmel proteins were present in the melanosomes of human melanoma cells. Targeting ?-synuclein

Research 98

Research Protein In-Vitro Gene Therapy 98

XTalks

APRIL 23, 2021

dMMR occurs when a cell is unable to repair errors made during cellular replication, owing to mutations in DNA mismatch repair genes such as MLH1 or MLH2 , resulting in high-frequency microsatellite instability (H-MSI).

FDA Approval 105

FDA Approval Trials Antibody DNA 105

pharmaphorum

JANUARY 27, 2023

She knows only too well how understanding the importance of patient centricity is crucial to the success of any modern life sciences company – but how has the landscape changed since 2019? “I The ABPI report also contained recommendations regarding this. I still feel that it is very important,” Dr Mullen said. “We’ve

Clinical Trials 103

Clinical Trials Clinical Trials Trials Development 103

XTalks

APRIL 26, 2021

This is because there are thousands of genes in malaria parasites compared to only about a dozen or so in coronaviruses. The vaccine is protein-based and contains an adjuvant (MM), a component that helps generate a stronger immune response, that was developed by Novavax. Malaria Vaccine Phase II Trial.

Vaccination 105

Vaccination Vaccine Immune Response Trials 105

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

APRIL 15, 2021

From a total of 30 single-nucleotide polymorphisms (SNPs) in genes previously identified as being associated with blood pressure, many had a relatively neutral effect on the risk of coronary artery disease. Figure 2: Using drug-target MR to validate sympathetic nervous system genes involved in heart failure.

Development 98

Development Drugs Genome Genomics 98

XTalks

NOVEMBER 7, 2022

Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. The first option is to design a personalized small NGS gene panel based on the somatic mutations that were detected in the patient, and this can be used for monitoring disease progression.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Drug Discovery World

AUGUST 10, 2022

For example, one clinical trial examined the safety and efficacy of a CAR T cell that includes a suicide gene, which produces a toxic protein that causes the CAR T cells to die upon exposure to a drug 10. If the gene does not integrate into the genome, the cell will not become a CAR T cell.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

Pharmaceutical Technology

AUGUST 31, 2022

ILC Dover, specialists in the innovative design of biopharmaceutical manufacturing solutions, continuously monitors the market in order to provide in-demand equipment such as flexible containment solutions that reduce bottlenecks by providing closed, Grade-A, aseptic environments for fill and finish activities.

Packaging 130

Packaging Packaging Production Containment 130

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

NOVEMBER 10, 2020

How many genes need to be scanned in order to determine the presence of a genetic disease? HS: Due to the heterogeneity of the rare disease population, there are many relevant gene lists, and the genomic data analysis of these patients covers almost 2,000 different genetic targets.

Genome 98

Genome Genomics Medicine Genetic Disease 98

XTalks

MAY 15, 2023

This condition arises from mutations in the GAL gene at Xq22, leading to the accumulation of glycosphingolipids within lysosomes in various tissues and organs, such as the heart, kidneys, eyes, vascular endothelium, nervous system and skin. Genetic confirmatory testing is then conducted to identify the specific mutations in the GLA gene.

FDA Approval 98

FDA Approval Gene Genetics Clinical Trials 98

XTalks

AUGUST 2, 2023

It contains the antiviral medications nirmatrelvir and ritonavir. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Comirnaty was first approved by the US Food and Drug Administration (FDA) in August 2021 for individuals over the age of 16. billion in 2022. Both are anti-CD20 agents.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

XTalks

DECEMBER 13, 2023

Initial data is expected to be available later this year, and Alpine Immune Sciences is actively planning a comprehensive and expedited development program for povetacicept across multiple indications in 2024. This unique formulation has the ability to reduce components of cellular inflammation and foster tissue healing.

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

MARCH 11, 2021

Speaking at the virtual Capital Markets Day on Wednesday, CEO Werner Baumann emphasized Bayer’s long-term growth perspectives: “Bayer is a leading life science company, uniquely positioned at the intersection of health and nutrition – with attractive prospects for growth, earnings and cash flow.”

Sales 52

Sales Gene Therapy Marketing Gene 52

Drug Discovery World

JULY 15, 2022

The new sequences contain largely highly repetitive DNA structures such as centromeres, telomeres, acrocentric genomic regions, where massive arrays of tandem repeats predominate, and heterochromatin. In turn, this will aid our understanding of the cells capacity to silence harmful DNA elements or prevent inappropriate gene expression.

Genome 52

Genome Genomics DNA Containment 52