Development, Genetic Disease, In-Vivo and Trials

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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.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo

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Trending Sources

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

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

Gene Editing Gene In-Vivo Clinical Trials

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

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. Those funds were used to advance its discovery and development programs.

In-Vivo

In-Vivo Marketing Engineer Genetics

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

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

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

Gene Editing Gene In-Vivo Genetics

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Delveinsight

JANUARY 12, 2021

Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. million after Birinapant successfully becomes a part of the Phase I trials. BeiGene, Novartis partner to develop cancer drug Tislelizumab, an Anti-PD-1 Antibody.

Licensing

Licensing Licensing Antibody Gene Therapy

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

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.

Gene Therapy

Gene Therapy Gene Immune Response In-Vivo

Lysogene Reports Positive Biomarker Data With LYS-SAF302

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).

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials In-Vivo

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

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

Clinical Trials Clinical Trials Gene Therapy Trials

ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

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.

Gene Editing

Gene Editing Gene In-Vivo Genetics

BioSpace Movers & Shakers, Oct. 16

The Pharma Data

OCTOBER 15, 2020

The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. The committee is tasked with supporting BeyondSpring’s business development activities related to its lead asset, Plinabulin, and other pipeline assets. BeyondSpring – BeyondSpring Inc.

In-Vivo

In-Vivo Business Development Manufacturing Development

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

XTalks

OCTOBER 1, 2020

And although academia and industry have often been pitted against each other in the realm of scientific discovery and development, there may be more similarities and synergies between the two than we may think. WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation.

Scientist

Scientist In-Vitro Research Life Science

Advances in neuroscience drug discovery

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

Drugs Gene Clinical Trials Clinical Trials

Clinical Research Informer

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

Bayer trumpets $1bn CRISPR deal with Mammoth Bio

Trending Sources

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion

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

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

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

Lysogene Reports Positive Biomarker Data With LYS-SAF302

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

BioSpace Movers & Shakers, Oct. 16

Transitioning Between Academia and Industry: Advice from Leading Scientists That Made the Switch

Advances in neuroscience drug discovery

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