pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

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Gene Therapy Gene Marketing Genetic Disease 135

BioPharma Reporter

JULY 21, 2022

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.

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Genetic Disease RNA Genetics Regulation 52

pharmaphorum

NOVEMBER 27, 2020

The regulator has given a green light to use of Symkevi (tezacaftor/ivacaftor) with Vertex’ Kalydeco (ivacaftor) in patients ages six years and older who have two copies of the F508del mutation in the CFTR gene, or one F508del copy and one of 14 other so-called “minimal function” mutations in CFTR.

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Gene Genetic Disease FDA Approval Genetics 134

The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Genetics Containment Genetic Disease Gene 52

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. One chapter discusses how to form early partnerships with regulators.

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Development Gene Therapy Clinical Trials Clinical Trials 98

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.

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Drugs Genetic Disease Gene Therapy Trials 98

pharmaphorum

SEPTEMBER 6, 2021

The biotech said that the US regulator ordered the pause on the study after liver tumours were seen in mice given the therapy in preclinical testing. PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods.

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Gene Therapy Gene Genetic Disease DNA 94

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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FDA Approval Genetic Disease Genetics Clinical Trials 52

XTalks

SEPTEMBER 12, 2022

ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme. Related: FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures.

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FDA Approval Genetics Trials Gene 98

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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FDA Approval Genetics Trials Drugs 97

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.

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DNA Gene Gene Silencing Genome 98

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.

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Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. By gene editing the patient’s own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases,” he added.

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Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. These could feature in the next four to five years.

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Drug Delivery Vaccination Vaccine Research 147

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. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning.

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Drugs Clinical Trials Clinical Trials Genetic Disease 52

XTalks

JANUARY 3, 2025

FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism.

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FDA Approval Drugs Clinical Trials Clinical Trials 59

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

MARCH 16, 2021

in patients with VHL disease-associated RCC. Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. If not properly regulated, the accumulation of HIF-2?

Drugs 52

Drugs Trials Genetics Protein 52

The Pharma Data

SEPTEMBER 22, 2020

All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death. Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two.

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Clinical Development Gene Therapy Development Gene 52

XTalks

SEPTEMBER 16, 2024

HAE is a rare genetic condition that causes episodes of severe swelling in various parts of the body, including the face, hands, feet and airway. HAE is caused by a deficiency or dysfunction of the C1-inhibitor, a protein involved in regulating inflammation.

Protein 52

Protein Genetics Trials Allergies 52

Delveinsight

FEBRUARY 11, 2021

Avrobio designed the Plato platform to optimize vector copy numbers, personalize the conditioning regimen, aid automated manufacturing and otherwise promote and industrialize its lentiviral vector-based approach to treating genetic diseases.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. Prevalence: 1 in 3,500.

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Trials Gene Hormones Clinical Trials 98

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

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.

DNA 52

DNA Scientist Genetic Disease Research 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

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

The Pharma Data

AUGUST 3, 2021

The use of a sham procedure in STEER is included to provide a comparison group for an unbiased collection and assessment of efficacy, safety and tolerability of OAV-101 IT for this older population where the disease progression is slower. Spinal Muscular Atrophy (SMA) ?

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Clinical Trials Clinical Trials Gene Therapy Gene 52

The Pharma Data

NOVEMBER 2, 2020

This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene ( FGFR3 ), a negative regulator of bone growth. BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. In the U.S.,

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Drugs Containment Trials Marketing 40

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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Protein Production Development Drugs 40

XTalks

OCTOBER 1, 2020

Sooter has also found success in industry, leading and bringing together the work of different teams of researchers at NeuBase to help drive the company’s goal of developing cures and treatments for genetic diseases, including Huntington’s Disease. She says, “It’s been a lot of fun; it’s a decision I’m glad I made.”.

Scientist 52

Scientist In-Vitro Research Life Science 52

XTalks

NOVEMBER 29, 2024

Govorestat is a central nervous system (CNS)-penetrant aldose reductase inhibitor designed to target a critical problem in classic galactosemia — a rare genetic condition that prevents proper metabolism of the sugar galactose. The lack of existing treatments amplifies the unmet need for a viable therapy.

Drugs 98

Drugs Compliance Clinical Trials Clinical Trials 98

XTalks

FEBRUARY 13, 2025

HAE is a rare genetic disorder characterized by recurrent episodes of severe swelling (angioedema) in various parts of the body, including the skin, gastrointestinal tract and airways. It is currently under FDA review and, if approved, would be the first oral, on-demand HAE treatment.

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Trials Genetics Allergies Drugs 82