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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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.

In-Vivo 52

In-Vivo Gene Editing 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.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

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.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

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.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

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. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

pharmaphorum

AUGUST 24, 2021

The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 diabetes and blood disorders sickle cell disease (SCD) and beta thalassaemia. If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52