pharmaphorum

AUGUST 12, 2020

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8 billion last year to $3.8

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Drugs Genetic Disease Gene Therapy Trials 95

pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.

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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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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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.

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Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Streamlining trial designs and regulatory pathways can help. Optimising these processes and exploring alternative delivery methods can bring down costs.

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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.

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FDA Approval Gene Clinical Trials Clinical Trials 52

XTalks

SEPTEMBER 12, 2022

Related: FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures. Sanofi picked up its first Xenpozyme approval for the treatment of Niemann-Pick from Japanese regulators in March followed by an approval from European officials in May. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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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.

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FDA Approval Genetics Trials Drugs 97

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

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.

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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.

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FDA Approval Gene Therapy Gene Clinical Trials 59

Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

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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.

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FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

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DNA Scientist Genetic Disease Research 52

Drug Discovery World

JULY 11, 2022

These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. The complexities of sample management can significantly extend the timelines for clinical trials. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10.

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pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on. .

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Gene Therapy Gene Genetic Disease Genetics 83

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. In the U.S., About BioMarin.

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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

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

Drug Discovery World

APRIL 30, 2024

At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed. The Mobilize trial is investigating the mRNA-4359 therapy and its potential for treating melanoma, lung cancer and other solid tumour cancers. We believe it can now do the same for rare diseases.” “We

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RNA Protein Vaccination Vaccine 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.

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Gene Editing Gene In-Vivo Genetics 52

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. 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.

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Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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XTalks

OCTOBER 1, 2020

The drug is currently in clinical trials for alcoholic hepatitis (AH), nonalcoholic steatohepatitis (NASH), as well as in COVID-19 patients with acute liver or kidney injury. Working on finding therapies for genetic diseases at NeuBase, Dr. Sooter says, “Everyday, we get up and that’s our goal and it really drives everyone.

Scientist 52

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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.

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