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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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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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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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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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. Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Tailored oncology.

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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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Pharmaceutical Technology

FEBRUARY 28, 2023

The vast majority of rare diseases have a genetic cause, with well-known examples including cystic fibrosis, sickle cell disease, Huntingdon’s, and various muscular atrophies and dystrophies. This often puts patients in difficult situations where they are passed between doctors until a correct diagnosis is reached.

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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. Reflecting on the evolution of rare disease treatments, Dr. Philipson emphasizes the critical role of understanding disease biology and genetics.

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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. Behind-the-scenes research lab videos showcasing drug development.

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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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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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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. Gene therapy can ultimately only take you into the liver,” he explains.

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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.

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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.

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Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Douglas Lee of Bayer Pharmaceuticals described this change as a major shift in how patients access healthcare and “a change in the mindset that you need to see the doctor for everything.”

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The Pharma Data

MAY 15, 2021

I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about gene therapy. Gene therapy is a huge deal – some gene therapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff. Yet lots of them don’t get HCT.

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XTalks

DECEMBER 20, 2023

The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy. Check out this article to learn about the biotech companies that went public this year.

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pharmaphorum

SEPTEMBER 1, 2020

Another military man in the drugs business was Edward Robinson Squibb, who as a naval doctor during the Mexican-American war of 1846–1848 threw the drugs he was supplied with overboard due to their low quality. This knowledge of genetics and the underlying cause of many diseases, including cancer, has resulted in powerful new drugs.

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