pharmaphorum

OCTOBER 8, 2020

Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies. Benjamin Oakes.

Gene Editing 56

Gene Editing Gene In-Vivo Clinical Trials 56

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Rare diseases can often be progressive, chronic and fatal. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

DECEMBER 9, 2020

The new company, created by Hillhouse Capital Venture Partners, launched this week with a mission to pursue the most promising advances in medicine for underserved patients in Asia and around the world. A solution could be at hand with Overland Pharmaceuticals. Below is a roundup of some of this week’s interesting news.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. Li is a prominent neuroscientist and former professor at Johns Hopkins Medicine.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

Drug Discovery World

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . billion, compared to $19.9 million price point per dose. Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

DECEMBER 12, 2023

AATD patients face a critical unmet need given the limited effectiveness of current standard-of-care options for either the liver or the lung manifestations of the disease,” said Dr Jeffrey Teckman, an expert in AATD from St Louis University School of Medicine and a long-time advisor to Korro.

RNA 52

RNA In-Vivo Genotype Genetics 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

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 Trials 52

Drug Discovery World

NOVEMBER 9, 2023

Peter O’Callaghan, PhD, Head of Expression System Sciences, Biologics and Licensing, Lonza, on: ‘Solve bispecific heavy-light chain mispairing with bYlok technology’. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

Engineer 52

Engineer Antibody Protein In-Vivo 52

The Pharma Data

JULY 8, 2021

Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Despite recent advances in severe asthma, many patients may not qualify for or respond well to current biologic medicines.(2-5) Reese, M.D., About Severe Asthma.

Allergies 52

Allergies Trials In-Vivo Medicine 52

Drug Discovery World

FEBRUARY 26, 2024

For example, Vertex Pharmaceuticals’ stem-cell treatment for sickle-cell disease and transfusion-dependent β-thalassemia was authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) in November 2023, making it the first licensed therapy that uses the gene-editing tool CRISPR to help treat patients.

Manufacturing 52

Manufacturing In-Vivo Development Production 52

The Pharma Data

OCTOBER 15, 2020

The scientific advisory board is led by Kai Zacharowski, director of the Department of Anesthesiology, Intensive Care Medicine & Pain Therapy at the University Hospital Frankfurt, and is currently president of the European Society of Anesthesiology. BeyondSpring – BeyondSpring Inc. At Spark, he served as head of U.S.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In million deaths worldwide” 1.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59