Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. DDW Multimedia Editor Megan Thomas explored three trends in the market.

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

pharmaphorum

JULY 13, 2021

The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system. All told, the deal could be worth up to $1.2

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

In-Vivo 80

In-Vivo FDA Approval Gene Gene Editing 80

Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. He holds more than 200 patents related to AAV technology.

Gene Therapy 52

Gene Therapy Gene FDA Approval Clinical Trials 52

XTalks

MARCH 28, 2024

Early clinical trials have shown promising results and this therapy could become a valuable addition towards CRC treatment in the future. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

The Pharma Data

DECEMBER 27, 2020

This year has been difficult, but it has reinforced the importance of our mission: to treat and potentially even reverse the effects of serious diseases and conditions by advancing our novel cell therapy product candidates through clinical trials and into the hands of physicians.

Manufacturing 52

Manufacturing Production Development Clinical Trials 52

XTalks

AUGUST 2, 2023

Paxlovid was first authorized under the FDA Emergency Use Authorization in December 2021 ; however, it has received FDA approval on May 25, 2023. pneumoniae serotypes) replaced the company’s first pneumococcal conjugate vaccine Prevnar (PCV7, approved by the FDA in February 2000) in a February 2010 FDA approval.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies.

RNA 109

RNA Vaccination Vaccine Pharma Companies 109

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. In 2021, we saw a number of innovations in fields like neuromodulation, with several neurostimulation devices having received FDA approvals.

Life Science 98

Life Science RNA Vaccination Vaccine 98

pharmaphorum

NOVEMBER 2, 2021

In 2019, there were 57 bsAb candidates in clinical trials, against both haematological and solid tumours. In the pipeline , Amgen has BiTE molecules targeting seven types of cancer in clinical trials, as well as further structures in the design stage. Simplified schematic overview of the proposed mechanisms of action for bsAbs.

Antibody 52

Antibody Immune Response Production Engineer 52

Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccination Vaccine Gene 111