The Pharma Data

FEBRUARY 22, 2022

As part of its on-going work to track variants, WHO’s Technical Advisory Group on SARS-CoV-2 Virus Evolution ( TAG-VE ) met yesterday to discuss the latest evidence on the Omicron variant of concern, including its sublineages BA.1 1 in its genetic sequence, including some amino acid differences in the spike protein and other proteins.

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pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

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XTalks

JUNE 28, 2023

DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. In its approval, the FDA said it considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease for these children and the urgent unmet medical need.

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Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Other Challenges: Limited insurance coverage: Many insurance companies are hesitant to cover CGTs due to their high price tags and limited long-term data.

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pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. million price tag. Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8

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The Pharma Data

MARCH 23, 2022

Unfortunately, in a Phase 3 clinical trial, treatment with remdesivir did not make a difference in mortality for Ebola virus disease patients. Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material.

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XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. With a price tag of over $2.5 While SMN1 gene mutations cause the disease, the number of copies of the SMN2 gene modifies the severity and helps determine the type of the condition.

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Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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The Pharma Data

OCTOBER 21, 2020

The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). Traditionally, Ph-positive ALL had a poor prognosis, but in the past two decades that has changed with the introduction of drugs that target the genetic anomaly. © 2020 HealthDay.

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The Pharma Data

OCTOBER 21, 2020

The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). Traditionally, Ph-positive ALL had a poor prognosis, but in the past two decades that has changed with the introduction of drugs that target the genetic anomaly. © 2020 HealthDay.

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pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S. CellCentric, a U.K-based

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pharmaphorum

AUGUST 18, 2022

.” The gene therapy specialist’s chief executive Andrew Obenshain said the approval “marks a watershed moment for the field of gene therapy” and is “ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

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pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Zolgensma was backed for type 1 SMA in draft guidance issued last month, even though the gene therapy’s £1.79

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pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS). million in Europe.

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Roots Analysis

JANUARY 28, 2024

Velocity: Big Data needs to be handled promptly as businesses generate data at an unprecedented speed, driven by the growing adoption of Internet of Things (IoT), and technologies, such as RFID tags, sensors, and smart meters are used to deal with the high velocity of data in real-time. It is loosely arranged into categories using meta tags.

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XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​.

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XTalks

AUGUST 17, 2020

To learn more about innovations in the field, Xtalks spoke to Anish Suri, PhD, president and chief scientific officer of Cue Biopharma about the company’s initiatives in the development of immune-based biological compounds for the treatment of cancers and autoimmune diseases. cells, receptors) to treat disease.

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XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5 Hemophilia B primarily affects men.

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XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

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pharmaphorum

DECEMBER 7, 2021

Scientists in the US have found that people who use Viagra for erectile dysfunction seem to have a lower risk of developing Alzheimer’s disease – a link that they say should be examined more closely in clinical trials.

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Delveinsight

DECEMBER 3, 2020

Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences. The team said that if further studies turn out to be satisfactory, they could develop therapies, which employ the same method for repairing damage in other organs and probably treat age-related diseases in humans too.

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Drug Discovery World

NOVEMBER 9, 2023

Advances in discovery and engineering of antibodies for non-cancer targets This session will feature a range of presentations which include: Joshua Tan, PhD, Chief, Antibody Biology Unit, National Institute of Allergy and Infectious Diseases, National Institutes of Health, on: ‘Discovery of broadley-neutralising antibodies against Coronaviruses’.

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