pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

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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.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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Clinical Trials Clinical Trials Gene Therapy Gene 52

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

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XTalks

DECEMBER 13, 2023

Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Sickle cell disease is a group of inherited red blood cell disorders that affect approximately 100,000 people in the US, most of whom are Black. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy Gene FDA Approval In-Vivo 98

Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

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