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

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

NOVEMBER 10, 2023

Treatments for cTTP includes prophylactic plasma-based therapy for individuals with chronic disease to reduce the risk of clotting/bleeding by restoring the low amounts or absence of the ADAMTS13 enzyme. For prophylactic ERT, Adyznma is given to help reduce the risk of disease symptoms.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

OCTOBER 18, 2020

This study is the first to show that by increasing NAD + , NR helped protect telomeres which are important in addressing life-threatening telomere-related diseases, paving the way for future clinical research. The role of ChromaDex in this study was to provide NR through the ChromaDex External Research Program (CERP).

DNA 52

DNA Scientist Genetic Disease Research 52

Delveinsight

AUGUST 17, 2021

The efficacy and safety results were produced from a broad research study, multicenter, open-label, randomized phase 3 trial data from the Keynote-581 trial of the first-line therapy indicated to the patients that resulted in an extension of their survival. The rarity of disease – 1.36 person/million around the globe.

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

The Pharma Data

NOVEMBER 2, 2020

President Worldwide Research and Development at BioMarin. “We advocate for rigorous scientific and medical research to improve the quality of medical care for little people.” ” Growing Stronger’s mission is to improve the quality of medical care for people through supporting research. and Europe.

Drugs 40

Drugs Containment Trials Marketing 40

The Pharma Data

NOVEMBER 11, 2020

Research and Development (R&D) Expenses – R&D expenses were $25.2 We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies. Inozyme expects to share data from this study in 2021. H1 2021: Initiation of Phase 1/2 clinical trial.

Clinical Trials 40

Clinical Trials Clinical Trials Trials Development 40

pharmaphorum

JANUARY 27, 2023

Appropriate site selection [is] really important to engage with enthusiastic investigators that want to be involved in pioneering research and innovations.” The ABPI report also contained recommendations regarding this. Also,” she continued, “they [must] have the right access to the right patients to enrol in the study.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Development 105

pharmaphorum

SEPTEMBER 9, 2020

Charlie has spent more than 26 years working on the human genome – 22 of which were spent at the Wellcome Sanger Institute, helping to assemble the human genome and trying to understand the function of the genes contained within it. Everything the project does has to be rubber-stamped by the patients,” he says. We have a very powerful voice.

Genome 86

Genome Genomics Genetics Genome Project 86

pharmaphorum

SEPTEMBER 9, 2020

Charlie has spent more than 26 years working on the human genome – 22 of which were spent at the Wellcome Sanger Institute, helping to assemble the human genome and trying to understand the function of the genes contained within it. Everything the project does has to be rubber-stamped by the patients,” he says. We have a very powerful voice.

Genome 73

Genome Genomics Genetics Genome Project 73

The Pharma Data

AUGUST 15, 2021

The WELIREG label contains a boxed warning that exposure to WELIREG during pregnancy can cause embryo-fetal harm. About Von Hippel-Lindau Disease. This is a rare genetic disease with an estimated incidence of 10,000 people in the U.S. As an inhibitor of HIF-2?, WELIREG reduces transcription and expression of HIF-2?

Hormones 52

Hormones Clinical Trials Clinical Trials Trials 52

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. The research was published earlier this month in the journal Cell.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccination Vaccine DNA 111

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. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Source link: [link] /.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

DECEMBER 3, 2020

December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. Translational Research / 2B. New preclinical data provide additional evidence of the potential for TANGO antisense oligonucleotides (ASOs) to provide a gene-specific, disease-modifying treatment for Dravet syndrome. 4, 2020 14:57 UTC.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

The Pharma Data

JULY 6, 2021

About SPK-8011 for haemophilia A Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. We currently have four programs in clinical trials.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

XTalks

APRIL 15, 2021

A 2015 study published in Nature Genetics found that the availability of human genetic data made investigational drugs twice as likely to pass pivotal trials and eventually be approved. Through this type of analysis, researchers are unable to say whether a drug target is specific to patients with HFrEF or HFpEF.

Development 98

Development Drugs Genome Genomics 98

The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. This press release contains forward-looking statements. About BridgeBio. Forward-Looking Statements.

Sales 52

Sales Marketing Containment Development 52

The Pharma Data

OCTOBER 28, 2020

GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

JANUARY 9, 2021

Boxer, MD Research Professor of Pediatrics and Communicable Diseases Department of Pathology Michigan Medicine at the University of Michigan and investigator in the Phase 3 study. President of Worldwide Research and Development at BioMarin. President of Worldwide Research and Development at BioMarin. About BioMarin.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 22, 2020

About Novartis Gene Therapies Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Disclaimer This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

Pharmaceutical Technology

APRIL 4, 2023

Orphacol® is a pharmaceutical product containing cholic acid, a substance found in the bile, which is used to digest fats. Orphacol® is used to treat adults and children who suffer from Bile Acid Synthesis Disorders (BASD), a genetic abnormality that makes them unable to produce bile.

Medicine 130

Medicine Marketing Production Genetics 130