Gene Therapy, Genetic Disease, Life Science and Trials

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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work?

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4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

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).

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Transcending expectations for cell & gene therapy development

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Bone marrow transplantation is also used in some infantile-onset cases to help delay disease progression.

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New White Paper on Advanced Therapies Sets up Developers for Success

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. Integrating patients into all trial functions. Collaboration.

Development

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Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

XTalks

NOVEMBER 27, 2023

Pompe disease primarily manifests as progressive muscle weakness, particularly affecting the skeletal muscles in regions such as the hips, legs, shoulders, arms and diaphragm. Sanofi also offers another ERT for Pompe disease known as Myozyme, which received FDA approval in 2006, and Lumizyme in 2010 for late-onset disease.

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Patient centricity and the changing pharmaceutical vista

pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

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Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.

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The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed. The Mobilize trial is investigating the mRNA-4359 therapy and its potential for treating melanoma, lung cancer and other solid tumour cancers. However, these therapies can cause severe adverse effects.

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The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Additionally, Parag V.

In-Vivo

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Clinical Research Informer

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Trending Sources

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

4 Life Sciences Trends for 2023

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

Transcending expectations for cell & gene therapy development

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

New White Paper on Advanced Therapies Sets up Developers for Success

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

Patient centricity and the changing pharmaceutical vista

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

The future outlook for mRNA therapies

BioSpace Movers & Shakers, Oct. 16

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