This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Doctors Gene Gene Therapy 
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.
286
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
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.
290 
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
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.
239 
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.
180 
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.
180 
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…
98 
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.
97 
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.
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.
105 
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. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? With a price tag of over $2.5
110 
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).
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.
97 
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.
145 
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.
98 
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 (..)
99 
Pharmaceutical Technology
JUNE 29, 2022
The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Further opportunities arise from the industry’s move towards sustainability.
295 
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).
58 
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 
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 
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.
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 
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 
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).
105 
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.
111 
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 
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 
World of DTC Marketing
JANUARY 31, 2021
Deloitte goes on to say “while biologics generally target smaller populations, “next-gen” therapies often target individual patients—sometimes as single treatments—resulting in truly personalized medicine. The cost is estimated to be in the billions.
187 
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 
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 
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 
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. Work to be done.
104 
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.
124 
WCG Clinical
JUNE 4, 2024
I am in the Doctorate in Nursing Practice program at the University of California, San Francisco, and I’m really looking forward to, becoming the first person in my family on either side to achieve a doctorate degree. We started experimenting with different gene therapies and antisense therapies.
52 
pharmaphorum
JUNE 1, 2022
Due to the pandemic, more people than ever had to turn to digital healthcare to achieve basic activities, such as speaking with a doctor or booking a visit with a healthcare professional. Spark Therapeutics developed a VR product , alongside a partner, to teach haemophilia patients about gene therapy. The training challenge.
145 
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 
pharmaphorum
JUNE 24, 2022
FoCus investigator Prof Karl Heinz Weiss of Salem Medical Centre Heidelberg said the results “have the potential to reframe the way doctors can think about the disease given that current therapies focus on removing copper from the blood.”
100 
Advarra
MARCH 10, 2023
These are reasons for biotech organizations to renew a focus on improving diversity in clinical research while bringing more sickle cell gene therapies to market. A visit to a podiatrist and family doctor yielded less than satisfactory diagnoses. Once, she developed a painful, raised lesion on her heel.
pharmaphorum
NOVEMBER 4, 2022
.” At Novartis, she was involved with projects including multiple sclerosis therapy Kesimpta (ofatumumab), migraine therapy Aimovig (erenumab), and Parkinson’s disease, and the acquisition by Novartis of optogenetic gene therapy company Vedere Bio.
59 
pharmaphorum
NOVEMBER 17, 2020
Especially if you move into things like gene therapies.”. With COVID-19 posing an added risk to people with CF, Vertex was “even more motivated” to secure its supply chain and access to drugs. Engaging with the health system as early as possible and working flexibly was key to the success, he adds. “It It has to be more collaborative.
117 
The Pharma Data
DECEMBER 10, 2020
Dr. Pirozzi received a Doctorate of Medicine from Università Campus Bio-Medico di Roma in Italy, a Doctorate of Philosophy in Immunology from Sapienza Università di Roma in Italy, a Post-Doctorate degree in Immunology from the Institut Pasteur in Paris, France, and business training at the London Business School, UK.
52 
Pharmaceutical Technology
FEBRUARY 1, 2023
This would be preferable for patients as doctors must administer Synagis to patients five times per month during peak RSV season. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.
162 
pharmaphorum
APRIL 11, 2022
Janssen’s Catherine Taylor, vice-president, EMEA medical affairs, therapy area strategy, discusses the importance of systemic innovation across the healthcare system to realise the full value of medicines. Medicines and vaccines are among the most powerful interventions that can help improve quality of life for people across the world.
115
Expert insights. Personalized for you.
We have resent the email to
Are you sure you want to cancel your subscriptions?
Let's personalize your content