XTalks

DECEMBER 13, 2023

In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia. The company has been facing financial woes despite the big approvals. Vertex-CRISPR’s Casgevy has a US list price of $2.2

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Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

JUNE 8, 2021

With a price tag of over $2.5 Recent data from the ongoing START phase I clinical trial, published in JAMA Neurology , supports the long-term favorable safety profile of Zolgensma in children aged up to six years with type I SMA. million, Zolgensma is currently the world’s most expensive drug. Zolgensma Gene Therapy: START Trial.

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Gene Therapy Gene Protein Trials 110

XTalks

OCTOBER 12, 2022

For ASL MR perfusion, water molecules in arterial blood from a feeder artery are magnetically tagged and tracked until they reach the region of interest. Then, the signal difference in the region of interest between the tagged images and control images can be computed to obtain cerebral blood flow (CBF) values ( Figure 3 ).

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Clinical Trials Clinical Trials Trials Drugs 93

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). It is specifically indicated to slow the progression of neurologic dysfunction in boys four to 17 years of age with early, active CALD. Related: Bluebird’s $2.8M

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FDA Approval Gene Therapy Drugs Gene 52

XTalks

FEBRUARY 27, 2024

Skyclarys (omobaloxone) was approved in 2023 for the treatment of adults and pediatric patients 16 years of age and older with Friedreich’s ataxia, a rare, neurological disease​​. With the approval, Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy also became the first CRISPR/Cas9-based therapy approved in the US.

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Research Drugs Gene Therapy Clinical Trials 59

Pharmaceutical Technology

JANUARY 27, 2023

James George, a research professor at the University of Oklahoma Health Sciences Center, said Cablivi “adds a new aspect to treatment, the prompt recovery of platelets and prompt resolution of transient neurologic symptoms”. In 2022, the number of risk sharing agreements peaked with 144 agreements being made in the pharmaceutical industry.

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Manufacturing Drugs Research Analyst FDA Approval 130

XTalks

NOVEMBER 24, 2022

Prior to Hemgenix, bluebird bio’s recently approved gene therapy Skysona for the rare neurological disorder cerebral adrenoleukodystrophy (CALD) held the record for the highest-costing drug in the world at $3 million and before that, it was Novartis’ one-dose gene therapy Zolgensma which is priced at about $2 million.

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Gene Therapy Gene FDA Approval Genetics 52

XTalks

AUGUST 23, 2021

Sperling, MD, a neurologist at Massachusetts General Hospital, and professor of neurology at Harvard Medical School. With a $56,000 price tag for a year’s worth of treatment, Aduhelm is far from the most expensive drug on the market. This is another case in which careful clinical trial design is of the utmost importance.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Drugs 52

XTalks

DECEMBER 21, 2022

Skysona (elivaldogene autotemcel) to slow the progression of neurologic dysfunction in boys four to 17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Several neurological stimulation devices with AI features were approved by the FDA this year. Manufacturer: CSL Behring LLC. Manufacturer: bluebird bio, Inc.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

pharmaphorum

FEBRUARY 26, 2021

Today, Janssen is one of the world’s leading research-based pharma companies and markets prescription medicines in the areas of gastroenterology, women’s health, mental health, neurology and HIV/AIDS, to name a few. “Dr For all the latest pharma news on Johnson & Johnson, follow pharmaphorum’s J&J and Janssen tags.

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Sales Production Pharma Companies Marketing 126

Drug Discovery World

NOVEMBER 13, 2023

Jens Fischer, PhD, Program Manager, Large Molecule Research Therapeutic Modalities, Roche Pharma Research and Early Development (pRED), on: ‘ Bispecific and next-generation antibodies for non-oncology indications such as ophthalmologic and neurologic diseases’.

Protein 59

Protein Antibody Engineer Scientist 59

The Pharma Data

MAY 4, 2021

Panzyga (immune globulin intravenous [human] ifas 10% liquid preparation) — In February 2021, Pfizer announced that the FDA approved the supplemental Biologics License Application (sBLA) for Panzyga to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP).

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Vaccination Vaccine Production Sales 40

Pharma Marketing Network

JUNE 8, 2020

I mean, you know, there has obviously been a lot of breakthrough biologics and different therapeutics like around cancers and things like that, that have high price tags because maybe they don’t have as big a market as you know, something’s wrong, you know blood pressure medication. So yeah, there is plenty to talk about there.

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HR Marketing Doctors Doctor 40