pharmaphorum

NOVEMBER 12, 2021

It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest. The post AI tool may help doctors select best drugs for COVID patients appeared first on.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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pharmaphorum

JANUARY 29, 2021

Four million UK patients could benefit annually from genetic testing before being prescribed common medicines, according to new research. The goal was to see how many patients are started on new prescriptions each year that could be potentially optimised by genetic testing. Estimating the potential impact of implementing pre?emptive

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pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

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FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials. One family’s daughter was the first U.S.

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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Scienmag

JANUARY 26, 2021

Credit: Photo credit: Charlie Ehlert/University of Utah Health In 2012, four-year-old Bertrand Might became the first-ever patient diagnosed with a rare genetic disorder called N-glycanase (NGLY1) deficiency. The discovery of this condition and Bertrand’s diagnosis allowed doctors to look for other children with the same genetic defect.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.” .

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Gene Editing Gene Gene Therapy Genetics 98

Scienmag

JUNE 29, 2021

Scientists and doctors at University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH) have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS).

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Gene Therapy Gene Doctors Doctor 58

STAT News

APRIL 3, 2023

They had been modified in a lab by adding a functional gene to compensate for a defective one. Conner’s doctors expect that this groundbreaking gene therapy, which costs a staggering $3 million a patient, will stave off a fatal degenerative brain disease and save his life.

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pharmaphorum

NOVEMBER 3, 2020

Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet despite huge leaps forward in gene sequencing in recent years, diagnoses are going down instead of up. There are no clinical practice guidelines, meaning doctors treat on a case-by-case basis. Raising awareness, offering support.

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Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. Continuing advancements within this field deliver new hope to doctors and patients, transforming disease outcomes for previously incurable indications. And it’s within the family – Novozymes!”. This venture is a perfect match; we complement each other.

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pharmaphorum

SEPTEMBER 21, 2021

In other cases, rare CNS conditions may be passed on from one generation to the next without knowledge or understanding that the reason for suffering or death among previous generations was genetic in basis and therefore identifiable, and perhaps even treatable. Doctors are often unfamiliar with these conditions. About the author.

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Gene Therapy Gene Clinical Trials Clinical Trials 90

The Pharma Data

DECEMBER 28, 2020

The Corps is a group of doctors, public health professionals and community advocates working to reverse the COVID-19 outbreak and protect our city. About Fulgent Genetics. TEMPLE CITY, Calif., It ensures that anyone with the virus receives care and can safely isolate to prevent the spread. For more information, visit [link].

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Drug Discovery World

NOVEMBER 27, 2023

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Production Engineer Gene Editing Gene 59

The Pharma Data

JANUARY 21, 2021

Studies Pinpoint Genes That Raise Risk for Breast Cancer. New research into exactly which genetic mutations increase breast cancer risk in women without a family history of the disease may help guide decisions about screening and preventive surgery, doctors say. She was not involved in the new studies.

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Gene Vaccination Vaccine Genetics 52

pharmaphorum

FEBRUARY 16, 2022

She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32

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Drug Discovery World

MAY 29, 2024

Marc Hummersone, Senior Director of Research and Development (R&D) at Astrea Bioseparations, shares insight on the challenges and opportunities in cell and gene therapy (CGT) with DDW’s Megan Thomas. That takes a lot of effort, and some very clever people to get that amount of genetic information into a single plasmid DNA.

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Gene Therapy Gene Genetics DNA 109

XTalks

NOVEMBER 18, 2021

“The first time I heard the term hypertrophic cardiomyopathy, I was in the doctor’s office in the room getting diagnosed. HCM is a genetic heart condition in which the muscle of the heart thickens, causing it to work harder to pump blood. Through genetic testing, it was revealed that Juanea carries the gene for the heart condition.

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Doctors Doctor Genetics Gene 98

Bioengineer

JULY 22, 2021

Geneticists from Trinity College Dublin have discovered how a specific genetic mutation called H3K27M causes a devastating, incurable childhood cancer, known as diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reverse its effects to slow cancer cell growth with a targeted drug.

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pharmaphorum

AUGUST 13, 2020

NS Pharma’s Viltepso (viltolarsen) has been approved in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD.

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FDA Approval Drugs Gene Trials 52

pharmaphorum

SEPTEMBER 9, 2020

Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate. He is currently the patient advocacy and engagement lead at Congenica, a digital health company that is investigating the genetic basis of rare diseases. For some people, a job is more than just a job.

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XTalks

APRIL 6, 2022

Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. If that happens, it is a big setback for patients and the doctors treating them,” said Dr. Rosenfeld in a news release by Cancer Research UK. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

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DNA In-Vitro Doctors Doctor 105

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

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Genetic Disease Protein RNA Doctors 104

XTalks

SEPTEMBER 22, 2020

“Helping people understand and manage their heart health has always been a priority for Fitbit, and our new ECG app is designed for those users who want to assess themselves in the moment and review the reading later with their doctor,” Eric Friedman, Fitbit co-founder and CTO said in a statement.

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pharmaphorum

AUGUST 11, 2022

Pharmacogenomics, the understanding of how genes impact an individual’s response to medications, provides a potential outlet to better optimised therapies. The participants undergo monitoring for genetic variations that could potentially negatively impact their reaction to therapies. About the Author.

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Genome Genomics Genetics Drugs 98

pharmaphorum

SEPTEMBER 9, 2020

Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate. He is currently the patient advocacy and engagement lead at Congenica, a digital health company that is investigating the genetic basis of rare diseases. For some people, a job is more than just a job.

Genome 80

Genome Genomics Genetics Genome Project 80

pharmaphorum

JUNE 24, 2022

Wilson disease is a rare and progressive genetic condition in which the body’s process for removing excess copper is broken, allowing it to build to toxic levels and damage the liver, brain and other vital organs. Now, the data has been revealed – and according to investigators could transform treatment.

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Drugs Gene Therapy Compliance Trials 100

The Pharma Data

SEPTEMBER 1, 2021

Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. “We used CRISPR technology to reprogram the genes in stem cells. million adults in the United States. Simon Professor of Orthopaedic Surgery. ” The new findings are published online Sept.

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pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The disease is also difficult to diagnose and often takes around six years before doctors correctly identify it.

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FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

FEBRUARY 28, 2022

Many of the small challenges that face individual doctors and patients in seeking to find a solution for a rare disease also contribute to the difficulty of researching and developing an effective treatment. Once diagnosed, only 5% of rare diseases actually have an effective treatment. The outlook for rare disease R&D.

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Gene Therapy Gene Development Medicine 104

XTalks

MAY 17, 2024

Dr. Philipson’s transition to the biotech sector began with a position at UK-based biotech Trizell, where his efforts culminated in a gene therapy FDA submission for bladder cancer. Reflecting on the evolution of rare disease treatments, Dr. Philipson emphasizes the critical role of understanding disease biology and genetics.

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Development Drugs Clinical Trials Clinical Trials 52

The Pharma Data

MAY 9, 2022

Currently, after surgery, doctors examine a specimen of a tumor under a microscope and grade it one, two or three in its aggressiveness. The paper was published today in Nature Genetics. “Eventually we hope to tailor medical therapy to the genetic changes within each individual person’s meningioma,” Magill said.

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Drugs Scientist DNA Genetics 52

Scienmag

DECEMBER 22, 2020

Clinicians view autism as a broad spectrum of related disorders, and the origin of the disease’s heterogeneity has puzzled scientists, doctors, and affected families for decades. NEW YORK, NY (Dec. 22, 2020)–One of the most recognizable characteristics of autism is an amazing diversity of associated behavioral symptoms.

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Doctors Doctor Scientist Research 53

Pharmaceutical Technology

NOVEMBER 4, 2022

Through this work, a mutation was discovered in the lung cancer gene. And the oral treatment was highly effective for patients with this gene mutation, whereas the treatment was largely ineffective for lung cancer patients without the mutation. That has the potential to save the NHS huge amounts of money.”.

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