XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

XTalks

DECEMBER 4, 2024

Jotrol is positioned as a potential treatment for central nervous system (CNS) disorders, including Parkinson’s disease, Alzheimer’s disease and rare conditions such as Friedreich’s ataxia and mucopolysaccharidoses type 1.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

Pharmaceutical Technology

MAY 4, 2023

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for KALYDECO (ivacaftor) to treat children with cystic fibrosis (CF) aged between one and four months. CF is a rare, life-shortening genetic disease that is estimated to impact over 88,000 people across the globe.

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In-Vitro Genetic Disease Medicine Genetics 246

Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

Pharmaceutical Technology

OCTOBER 25, 2022

Astellas chief strategy officer Naoki Okamura said: “Gene therapy is the cornerstone of Astellas’ Primary Focus, Genetic Regulation; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

Fierce Pharma

JANUARY 10, 2024

In his first presentation as the new CEO of BioMarin, Alexander Hardy left no doubt about his mission for the rare and genetic disease specialist. | In his first presentation as the new CEO of BioMarin, Alexander Hardy left no doubt about his mission for the rare and genetic disease specialist.

Genetic Disease 88

Genetic Disease Genetics Drugs 88

BioSpace

SEPTEMBER 25, 2023

Ionis plans to submit a New Drug Application to the FDA. The company’s olezarsen cleared a late-stage study, eliciting a sharp reduction in triglyceride levels in patients with familial chylomicronemia syndrome.

Genetic Disease 87

Genetic Disease Genetics Drugs 87

Fierce Pharma

MARCH 20, 2024

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,

Gene Therapy 101

Gene Therapy Gene Genetic Disease Genetics 101

Medical Xpress

MARCH 6, 2023

The movement disorder ADCY5-related dyskinesia can be treated with the asthma drug theophylline. This has been shown in a recent study by Martin Luther University Halle-Wittenberg (MLU), University Medicine Halle and University of Leipzig Medical Center.

Genetic Disease 72

Genetic Disease Genetics Research Medicine 72

pharmaphorum

JULY 19, 2024

While the pace of biotech private rounds seems to have slowed a little of late, this week saw four big financings for Cardurion Pharma, Scorpion Therapeutics, CatalYm, and NGM Bio.Burlington, Massachusetts-based Cardurion led the quartet with a $260 million Series B for its heart-focused drug pipeline, which features two candidates in phase 2 testing (..)

Genetic Disease 105

Genetic Disease Genetics Drugs 105

World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

Life Science 225

Life Science Drugs Development Genetic Disease 225

BioSpace

JUNE 17, 2021

The FDA said that the drug, which has been studied and submitted for NPC indication, needs further data to support its use in this rare, progressive genetic disorder.

Genetics 52

Genetics Genetic Disease Drugs 52

Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

BioSpace

OCTOBER 22, 2023

The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.

FDA Approval 94

FDA Approval Genetic Disease Genetics Drugs 94

Pharmaceutical Technology

MAY 24, 2023

The AMP programme is a public-private partnership between a number of biopharmaceutical and life sciences companies, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH), along with non-profit and other organisations.

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Gene Therapy Gene Development Manufacturing 242

STAT News

MARCH 11, 2023

The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.

Genetics 98

Genetics FDA Approval Genetic Disease Clinical Trials 98

STAT News

FEBRUARY 3, 2023

The Boston drugmaker’s medicines for the genetic disease — which damages the lungs and other organs — have transformed the lives of patients, helping them abandon other time-consuming treatments, shrug off what would have been dangerous respiratory infections, and just live breathing easier.

Genetic Disease 98

Genetic Disease Genetics Medicine Drugs 98

pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

Drugs 98

Drugs Genetic Disease Gene Therapy Trials 98

pharmaphorum

MARCH 10, 2025

Beam says its base-editing drug has met its goals in a phase 1/2 trial in genetic disease AATD, and it wants to raise $500m to take it forward

Genetic Disease 66

Genetic Disease Trials Genetics Drugs 66

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

Gene Therapy 98

Gene Therapy Gene Drugs Gene Silencing 98

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The drug, which is administered as an oral liquid once a day after meals, costs almost $8,000 per bottle.

Drugs 98

Drugs Gene Therapy Genetic Disease Gene 98

Medical Xpress

JANUARY 20, 2023

The results are published in The Lancet Neurology.

Genetic Disease 76

Genetic Disease Genetics Clinical Research Drugs 76

XTalks

DECEMBER 18, 2024

Neurocrine Biosciences Crenessity (crinecerfont) has been given the green light by the US Food and Drug Administration (FDA) as a new treatment for congenital adrenal hyperplasia (CAH). Crenessity marks the third approved drug for Neurocrine. billion in revenue by 2024. to help build a community of CAH patients.

Genetics 116

Genetics Hormones Trials Drugs 116

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

Scienmag

DECEMBER 17, 2020

Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases. A team from the […].

Genetic Disease 52

Genetic Disease Genetics Gene Drugs 52

pharmaphorum

OCTOBER 19, 2022

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data. The post Synlogic preps phenylketonuria drug for phase 3 after mid-stage win appeared first on.

Drugs 98

Drugs Bacterium Genetic Disease Sales 98

Pharmaceutical Technology

APRIL 6, 2023

GRIA disorder is a family of rare genetic diseases. He will further employ this diagnostic to select drugs for restoring patients’ normal functioning. Evidence suggests that the majority of these disease-associated variants produce underactive AMPARs.

Genetic Disease 130

Genetic Disease Genetics Gene Drugs 130

Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

pharmaphorum

NOVEMBER 27, 2020

Vertex introduced Kalydeco in 2012, and has since followed up with launches of two-drug combinations Orkambi (lumacaftor/ivacaftor) and Symkevi – known as Symdeko in the US – in 2015 and 2018, respectively. Together these drugs cover the CFTR mutations seen in around half of all CF patients.

Gene 134

Gene Genetic Disease FDA Approval Genetics 134

STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

Gene Editing 94

Gene Editing Genetic Disease Genetics Protein 94

XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. However, these drugs have shown limited effectiveness in treating HFpEF. Case Study: Genomic Approaches in Coronary Artery Disease Drug Development.

Development 98

Development Drugs Genome Genomics 98

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. That process is also due to complete before year-end, said Leschly.

Gene Therapy 135

Gene Therapy Gene Marketing Genetic Disease 135

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA 52

RNA Genetic Disease Drugs Protein 52

XTalks

FEBRUARY 24, 2025

Ioniss New Drug Application (NDA) for donidalorsen to HAE attacks in adult and pediatric patients 12 years of age is currently under FDA review with a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025.

Trials 59

Trials RNA Allergies Drugs 59

pharmaphorum

SEPTEMBER 17, 2020

AstraZeneca has bought rights to an orally-active drug that inhibits PCSK9, offering a patient-friendly alternative to current cholesterol-lowering therapies that have to be delivered via a needle. The post AZ buys oral PCSK9 cholesterol drug from Dogma Therapeutics appeared first on.

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Drugs Antibody Sales Branding 66