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

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

The Pharma Data

DECEMBER 13, 2020

Before EdiGene, she served as Head of Strategy and Partnership at Junshi Biosciences, in charge of pipeline development strategy focus on oncology, autoimmune and metabolic diseases along with external partnership. Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 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.

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Gene Therapy FDA Approval Gene In-Vivo 98

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

Delveinsight

FEBRUARY 11, 2021

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. Late in 2019, a Fabry patient in phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869). About Lysogene.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. They are due to enrol 45 apiece, and are scheduled to readout next year.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

The Pharma Data

AUGUST 3, 2021

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

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Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

OCTOBER 15, 2020

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. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi. marketing and diagnostics.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

Drug Discovery World

FEBRUARY 22, 2024

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59