pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.

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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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Gene Therapy Gene Clinical Trials Clinical Trials 65

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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Doctors Doctor Drugs Gene Expression 135

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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Gene Therapy Gene FDA Approval Antibody 98

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

pharmaphorum

SEPTEMBER 13, 2021

AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease gene therapy appeared first on.

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Gene Therapy Gene Sales Trials 96

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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Gene Therapy Gene FDA Approval Production 119

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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Gene Therapy Gene Trials Genetics 105

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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Gene Therapy Gene Trials Clinical Trials 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

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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Gene Therapy Gene Protein Trials 110

Drug Discovery World

MAY 22, 2023

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. There are questions regarding whether enough doctors are able to recommend advanced therapies, as well as where to send patients to receive treatment.

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Gene Therapy Gene Drugs Contamination 52

Medical Xpress

MAY 16, 2023

That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. Sometimes scientists arrive at new findings and discoveries by chance.

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In-Vitro In-Vivo Genetic Disease Genetics 98

BioSpace

NOVEMBER 16, 2021

After 32 years, gene therapy pioneer Guangping Gao, Ph.D., is finally seeing the fruits of the doctoral thesis research he started in 1989.

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Gene Therapy Doctors Doctor Gene 103

Scienmag

MARCH 24, 2021

to UD lab will build on breakthrough discovery of gene linked to eye disorders Credit: Photo by Evan Krape When University of Delaware doctoral student Sandeep Aryal took the initiative to learn additional research techniques to study proteins present in the eye, his work resulted in the discovery of a new gene, known as […].

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Gene Doctors Doctor Protein 94

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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Gene Therapy Nurses Gene Doctors 98

STAT News

APRIL 6, 2023

… A major obstacle looms for the drugs of the future – not enough doctors know how to administer them , The Wall Street Journal writes. Promising therapies for some diseases are administered through lumbar punctures, which are not a routine part of a doctor’s daily practice.

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Doctors Doctor Drugs Packaging 98

Drug Discovery World

JANUARY 17, 2023

The study, led by OSU Associate Professor of Pharmaceutical Sciences Gaurav Sahay, Oregon State doctoral student Marco Herrera-Barrera, and Oregon Health & Science University Assistant Professor of Ophthalmology Renee Ryals, was published in Science Advances. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

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Gene Editing Gene Gene Therapy Genetics 98

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. appeared first on.

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Gene Sequencing Gene Genome Genomics 117

Scienmag

JUNE 9, 2021

First study to show gene expression changes in divers with ‘the bends’ reveals key role of genes for inflammation and immunity For over a century, researchers have known about “the bends”, a serious condition affecting scuba divers. Doctors do not yet have a definitive test […].

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Gene Expression Gene Doctors Doctor 75

Drug Discovery World

OCTOBER 5, 2023

Mosaic mutations’ – genetic changes which appear in some cells in the body but not all – in two genes, GNAQ or GNA11, cause a spectrum of conditions including Sturge-Weber syndrome and Phakomatosis Pigmentovascularis with Dermal Melanocytosis (PPV-DM). The researchers examined 42 children with either Sturge-Weber syndrome or PPV-DM at GOSH.

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Genetics Research Gene Doctors 59

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. Further opportunities arise from the industry’s move towards sustainability.

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Drugs Gene Therapy Manufacturing Production 295

pharmaphorum

JANUARY 26, 2021

Under the leadership of CEO Vas Narasimhan, who took over in 2018, the company has focused on using digital technology to engage with both patients and doctors. Marie-France Tschudin, president of Novartis’s pharmaceuticals business, said digital technology had helped to improve productivity and its interactions with doctors.

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Doctors Doctor Sales Clinical Trials 111

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. “Our findings paint a complex picture – with many, dynamic factors driving how tumours respond to treatment.”

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Genetics Gene Hormones RNA 52

pharmaphorum

SEPTEMBER 21, 2021

Doctors are often unfamiliar with these conditions. Doctors are heavily reliant on the observations of parents or caregivers and are constrained by the ability of the patient to convey symptoms they are experiencing. To complicate matters further, CNS conditions are often very difficult to identify, characterise and treat.

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Gene Therapy Gene Clinical Trials Clinical Trials 81

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

DECEMBER 17, 2020

Doctor Johnson has had an inspiring career that encompasses several aspects of the pharmacy industry. In keeping with her pioneering spirit, she recently expanded into Pharmacogenomics (PGx) gene testing for patients. Doctor Johnson aspires to incorporate the latest technologies available in healthcare to care for her patients.

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Medicine Pharmacy Doctors Doctor 52

DM Clinical Research

DECEMBER 15, 2020

However, they do usually mean you should see a doctor. Patients with infection symptoms should see their doctor immediately. Periodic fever syndromes are usually caused by gene mutations. If you suffer from fevers with no known cause, then ask your doctor about these conditions. Talking to a Doctor.

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Doctors Doctor Clinical Research Gene 45

pharmaphorum

FEBRUARY 16, 2022

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 Sangamo was working on a treatment based on autologous (patient-derived) stem cells and T cells that were gene-edited to carry the CCR5?32

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Gene Editing Research Gene Genetics 69

DM Clinical Research

DECEMBER 15, 2020

However, they do usually mean you should see a doctor. Patients with infection symptoms should see their doctor immediately. Periodic fever syndromes are usually caused by gene mutations. If you suffer from fevers with no known cause, then ask your doctor about these conditions. Talking to a Doctor.

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Doctors Doctor Gene Clinical Research 40

XTalks

NOVEMBER 18, 2021

Through genetic testing, it was revealed that Juanea carries the gene for the heart condition. Juanea said she felt “really really bad” when she found out she was a carrier of a mutated gene involved in HCM, but then had to come to “grips” with the fact that “this was something beyond [her] control.”.

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Doctors Doctor Genetics Gene 98

pharmaphorum

OCTOBER 21, 2021

Many are still predicting multibillion-dollar sales for the drug, albeit with a longer runway, despite reservations about its clinical efficacy among doctors and payers. Analysts meanwhile have suggested that Aduhelm may start to pick up momentum if that review is positive.

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Drug Discovery World

NOVEMBER 1, 2023

At the moment, we are focused on gene editing tools, and specifically CRISPR nucleases, trying to design strategies that allow us to use them more safely and effectively. Biography: Credit: Jurgita Satkūnaitė Originally from the US, Stephen Jones earned his doctorate in Molecular Biology, Cellular Biology and Biochemistry at Brown University.

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Gene Editing Research Gene Genome 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

Pharmaceutical Technology

JULY 28, 2022

Roche’s Susvimo (ranibizumab sustained release formulation) follows a similar theme; patients who are implanted with Susvimo are expected to visit their doctor only twice a year to have the implant refilled so that the medicine can be delivered to their eyes continuously. The therapy received FDA approval in October last year.

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Marketing Gene Therapy FDA Approval Doctors 130

The Pharma Data

MAY 18, 2021

1 This damage to the DNA (often called a gene mutation) can cause cancerous tumors to form in the skin. 4 This means there are changes in a specific gene—the BRAF gene—that has caused cancer cells to form and grow 4. Using a small piece of the tumor, a gene test can detect BRAF mutations. ” Additional Resources.

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Nurses Doctors Doctor Gene 52

XTalks

JANUARY 17, 2024

There are many aspects of oncology drug development that are driven by the unique nature of the treatments being developed and the needs of the patients, their caregivers and the doctors that treat them. Morrison remarked that the latest ESMO was unique in its broad discussion scope, touching on various innovative treatments.

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