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Bio Pharma Dive
MARCH 18, 2025
The case may give doctors pause before prescribing the treatment in the future, some analysts wrote. A young man treated with Elevidys died of acute liver failure.
357 
Bio Pharma Dive
DECEMBER 8, 2020
Doctors on Tuesday got a more detailed idea of how the therapy works and who might be eligible to take it, as results from a late-stage trial were presented at ASH.
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Bio Pharma Dive
MAY 7, 2023
Patient advocates and doctors are anticipating an approval this month of a treatment they believe to be a breakthrough for a deadly disease. But it’s not clear how well the therapy really works, putting the FDA in a difficult position.
293 
Bio Pharma Dive
APRIL 7, 2021
David Davidson is leaving Bluebird bio after nearly a decade as the gene therapy developer's top doctor. His new home, Tessera, just raised $230 million to advance its "gene writing" research.
242 
Bio Pharma Dive
DECEMBER 13, 2023
Uptake of Casgevy and Lyfgenia may be slow despite their dramatic benefit, physicians said, citing complexities in treatment, manufacturing and reimbursement.
184 
Fierce Pharma
JUNE 29, 2023
After an initial rejection in 2020 and a review delay earlier this year, BioMarin’s Roctavian has finally got the FDA go-ahead to introduce a gene therapy for a not-so-rare disorder.
128 
Bio Pharma Dive
OCTOBER 10, 2024
Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.
184 
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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Fierce Pharma
DECEMBER 21, 2023
As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their CRISPR-based gene-editing therapy Casgevy, one doctor who treats sickle cell disease patients is already | As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their world-first CRISPR-based gene therapy Casgevy, one doctor (..)
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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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STAT News
JANUARY 24, 2023
Then, months later, doctors delivered gene therapy directly to her brain. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Read the rest…
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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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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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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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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.
105 
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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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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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.
295 
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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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.
100 
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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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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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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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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XTalks
NOVEMBER 15, 2024
You know how to protect against COVID and flu so ask your pharmacist or doctor about scheduling an RSV vaccine, too.” The commercial talks about RSV being a “highly contagious virus” and that people 60 years of age and older have a higher risk of being hospitalized from RSV, making vaccination against the virus important.
105 
XTalks
JANUARY 21, 2025
A doctor inserts the catheter through a small incision in a vein in the groin and guides it to the heart. The catheters tip is positioned at various locations within the heart, where the doctor uses a generator to deliver RF energy. Companion diagnostic for Pfizers hemophilia B gene therapy Beqvez (idanacogene elaparvovec-dzkt).
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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.
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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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 […].
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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pharmaphorum
DECEMBER 2, 2021
Whether catching up with relatives, attending a business meeting, or even visiting a doctor, it became commonplace to conduct this type of activity via a screen. According a Pew Research study, 81% of participants said they have used video calls to talk to others since the onset of the pandemic. Tapping into data.
145 
Medical Xpress
DECEMBER 2, 2022
Changes in a single gene open the door for harmful gut bacteria to set off the inflammation that drives Crohn's disease, according to a new study led by Weill Cornell Medicine and NewYork-Presbyterian investigators. These findings could one day help doctors better select targeted treatments for patients with this immune disorder.
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Camargo
JULY 30, 2021
In 1981, I got my doctorate degree in Clinical Pharmacology, but more importantly, my research was heavily involved in neonatal medicine, developing drugs there. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified.
Trialfacts
MAY 27, 2024
This study will use brain imaging, genetics, questionnaires, and other assessments to map the brain systems and genes that underlie risk and resilience for mental health problems. Click Here to Check Your Eligibility for this Study The post Get Involved in Understanding Your Brain and Genes appeared first on Trialfacts.
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.
90 
Pharmaceutical Technology
AUGUST 12, 2022
We have patients calling us saying, ‘I want to get into this site, but they say [it will be] three months before they can see me,’ and it's not because the doctors don't want to do it. Gene therapies, another area highlighted in the strategy, are also being explored in the ALS clinical landscape, says Cudkowicz.
246 
Trialfacts
MAY 27, 2024
Study Location Research Centre: Monash Biomedical Imaging Location: 770 Blackburn Road, Clayton 3168 Lead Doctor: Professor Alex Fornito Ethics Committee: This study has been reviewed and approved by the Monash University Human Research Ethics Committee (Project #12692) Background Attention, young men! Contents Background Why Participate?
52 
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.
122 
World of DTC Marketing
JANUARY 31, 2021
Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases. Then there are ever increasing health insurance and hospital costs at a time when less people are going to the doctor’s office because of COVID-19.
187 
pharmaphorum
OCTOBER 27, 2020
Novartis’ pharmaceutical sales recovered in the third quarter of this year after an earlier COVID-19 decline, as new drugs like gene therapy Zolgensma gathered pace. . Correcting for currency fluctuations, net sales were flat at $12.3
98 
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.”.
98 
pharmaphorum
SEPTEMBER 12, 2022
Across LabCorp , we innovate through science and technology, with access to approximately 2,500 [doctors] and PhDs and over 700 patients. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.
105 
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.
130 
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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