The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

FDA Approval 52

FDA Approval Genetic Disease Genetics Clinical Trials 52

Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

XTalks

DECEMBER 29, 2022

In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. It has also been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the US regulator for both SCD and TDT.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

Delveinsight

FEBRUARY 11, 2021

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

MARCH 16, 2021

inhibitor belzutifan (pronounced bell-ZOO-ti-fan), a novel investigational candidate in Merck’s oncology pipeline, for the potential treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), not requiring immediate surgery. in patients with VHL disease-associated RCC.

Drugs 52

Drugs Trials Genetics Protein 52

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

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. Daybue, also called trofinetide, is a tripeptide.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Defects in hamartin or tuberin lead to a loss of regulation of mTOR, resulting in aberrant differentiation and development, which causes the generation of enlarged cells, as seen in TSC brain lesions.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

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

Drug Discovery World

JULY 11, 2022

Complexities arise while trying to establish a standard, cost-efficient, and expert process for research and manufacturing. These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. Making CGT work. Regulatory requirements. Conclusion.

Gene Therapy 52

Gene Therapy Gene Development Production 52

The Pharma Data

NOVEMBER 2, 2020

Although the FDA did not identify any filing issues with the NDA, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. Regulatory Status in the U.S.

Drugs 40

Drugs Containment Trials Marketing 40

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

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. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

XTalks

OCTOBER 1, 2020

WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation. Lin has made several transitions throughout her career, from being a clinician at an academic center, to academic research, to heading business development in industry. Motivations for Transition. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. ARUK 3 adds that while the UK government increased funding in dementia research from £28.2m years it takes to complete an entire cancer clinical trial.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

OCTOBER 28, 2020

Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52