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.

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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 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. BioMarin said the majority of patients who received Roctavian received corticosteroids to suppress the immune system for the gene therapy to be effective and safe.

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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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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

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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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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Pharmaceutical Technology

JUNE 29, 2022

In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Continuing advancements within this field deliver new hope to doctors and patients, transforming disease outcomes for previously incurable indications.

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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.

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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.

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Clinical Research Research Gene Therapy Drugs 226

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

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

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

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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.

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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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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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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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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.

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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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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.

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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.”

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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.

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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.

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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.

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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. Through partnerships and internal innovation, LabCorp is utilising these advances in order to improve healthcare stakeholders’ experiences for better patient results.

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Pharmaceutical Technology

FEBRUARY 28, 2023

This often puts patients in difficult situations where they are passed between doctors until a correct diagnosis is reached. There are 7,000 known rare diseases, most of which non-specialist healthcare professionals have never encountered before. The stakes are high when launching an orphan drug product, however.

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pharmaphorum

MAY 10, 2022

And those doctors have been born with TikTok on the mind and they will not go with the communication from the previous millennium. Hutnik gave an example of her work with Spark Therapeutics to develop a product to teach haemophilia patients about gene therapy. The first Gen Z HCPs are reaching the practice.

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Velocity Clinical Research

OCTOBER 4, 2023

Matt Fisher, MD qualified from Bristol Medical School in 2015 and worked as a doctor in general and emergency medicine for five years in the NHS. The research I have conducted so far covers a plethora of innovative therapeutic areas, from gene therapy to CRISPR technology and I look forward to diversifying this expertise at Velocity.”

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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.

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WCG Clinical

JUNE 4, 2024

We started, you know, experimenting with different gene therapies and antisense therapies. Doctors actually like to write. Our foundation has, we have identified a lead gene therapy candidate. So when I began, you know, the FOXG1 Research Foundation with a group of parents, we really act like a mini biotech.

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pharmaphorum

JULY 4, 2022

A very important question was raised by the webinar attendee of the diversity and inclusion of patients in clinical research, and Dr Hackett commented: “A lot of the work has been done in our Paediatric & Rare Disease network, as well as our Cell and Gene Therapy network, we are tackling the issue of diversity in data.

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pharmaphorum

NOVEMBER 30, 2022

On the theme of continuing to advance and evolve meeting capabilities as well, Shawah noted that most doctors are not on LinkedIn, so it is difficult to be connected with reps. Veeva is equally ‘super excited’. Coming in April 2023, this will be part of the Engage license.

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Pharma Marketing Network

FEBRUARY 27, 2025

For example, a LinkedIn article on the future of gene therapy can position a brand as an authority in genetic medicine, attracting biotech investors, researchers, and prescribing physicians. Doctor-patient interviews on managing chronic conditions. Engage in LinkedIn groups to build relationships with physicians and researchers.

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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. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? Zolgensma Gene Therapy: START Trial.

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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.

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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. Philipson, underscoring a commitment that would shape his career’s trajectory.

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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.

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