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US FDA approves two therapies that can edit gene of sickle cell patients

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The United States Food and Drug Administration (FDA) has approved two gene-based treatments for sickle cell disease.

The US FDA, in an announcement published on Friday, said the “milestone” treatments are Casgevy and Lyfgenia.

Sickle cell disease is an inherited red blood cell disorder in which the body produces abnormal haemoglobin that causes the red blood cells to become sticky and hard, according to the US Centers for Disease Control and Prevention (CDC).

Casgevy was approved for people who are 12 years and older with recurrent vaso-occlusive crises.

According to the FDA, Casgevy utilises a type of novel genome editing technology — CRISPR/Cas9 — that can accurately edit DNA.

“Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology,” the FDA said.

“Patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology.

“CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut.

“The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery.

“In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.”

LYFGENIA

Lyfgenia was approved for people who are 12 years and older with sickle cell disease and a history of vaso-occlusive events.

According to FDA, Lyfgenia is a
cell-based gene therapy that uses a lentiviral vector for genetic modification.

“With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease,” the agency said.

“Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient.”

THE PROCEDURE

According to FDA, Casgevy and Lyfgenia are made from the patient’s own blood stem cells, which are modified and given back as a one-time, single-dose infusion as part of a
hematopoietic stem cell transplant.

“Prior to treatment, a patient’s own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia,” the agency said.

“Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.”

‘IMPORTANT MEDICAL ADVANCE’

Speaking on the approvals, Peter Marks, the director of FDA’s Center for Biologics Evaluation and Research, said the treatments represent an “important medical advance” in the deployment of innovative cell-based gene therapies.

Marks said the approvals follow “rigorous evaluations of the scientific and clinical data” to ensure safe and effective treatment.

Education

Canada now permits international students to work 24 hours

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Canada has increased the permissible work hours for eligible international students during academic terms from 20 to 24 hours per week.

This adjustment aims to provide greater opportunities for students to gain work experience while maintaining their focus on academics.

Marc Miller, Canada’s Immigration, Refugees, and Citizenship Minister, stressed the intent behind this policy change, saying, “This change will give students more flexibility to manage their time and gain practical experience.”

The decision builds on remarks Miller made in October 2022 when he emphasized the importance of work experience for international students.

At the time, he stated: “With the economy growing at a faster rate than employers can hire new workers, Canada needs to look at every option so that we have the skills and workforce needed to fuel our growth.

“Immigration will be crucial to addressing our labour shortage. By allowing international students to work more while they study, we can help ease pressing needs in many sectors across the country, while providing more opportunities for international students to gain valuable Canadian work experience and continue contributing to our short-term recovery and long-term prosperity.”

Key Changes to the International Student Program
Full-Time Work During Academic Breaks

International students can now work full-time during designated academic breaks, such as summer and winter holidays. These periods allow students to increase their earnings without interfering with class schedules, bolstering their financial stability while continuing their education.

The updated regulations permit students to work up to 24 hours per week during academic terms. This increase offers greater employment opportunities while ensuring students can maintain a sustainable study schedule.

Enhanced Reporting by Institutions

Designated Educational Institutions (DLIs) are now required to submit reports twice yearly to Immigration, Refugees, and Citizenship Canada (IRCC). These reports will confirm students’ enrollment status and academic progress, ensuring compliance with study permit requirements and upholding the integrity of the International Student Program.

Approval for Institution Transfers

Under the new rules, international students must obtain approval before transferring to a different DLI. This requirement ensures adherence to permit conditions and maintains academic standards across Canada’s institutions.

Special Provisions for Quebec Students

In Quebec, eligible international students can work off-campus without a separate work permit, provided they meet specific criteria. Students must be enrolled full-time in post-secondary, vocational, or professional training programs, or vocational training at the secondary level. These courses must be at least six months long and lead to a recognized degree, diploma, or certificate.

By introducing these measures, Canada aims to enhance opportunities for international students while addressing labour shortages in key sectors, ensuring a balance between work experience and academic success.

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Trump appoints Elon Musk to lead department of government efficiency

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US President-Elect Donald Trump has appointed Elon Musk, chief executive officer (CEO) of Tesla and SpaceX, to lead a soon-to-be-established Department of Government Efficiency (DOGE).

Musk, owner of X, the microblogging platform previously known as Twitter, was an avid supporter of Trump during the electioneering leading up to the November 5 presidential election.

The former president defeated Vice-President Kamala Harris after securing more than 270 of the electoral college votes required to win the poll.

A statement on Tuesday said Musk would run DOGE alongside Vivek Ramaswamy, a politician and entrepreneur.

It said the duo “will pave the way for my Administration to dismantle Government Bureaucracy, slash excess regulations, cut wasteful expenditures, and restructure Federal Agencies Essential to the ‘Save America’ Movement’.

“I look forward to Elon and Vivek making changes to the Federal Bureaucracy with an eye on efficiency and, at the same time, making life better for all Americans,” the statement quoted the president-elect as saying.

“Importantly, we will drive out the massive waste and fraud which exists throughout our annual $6.5 Trillion Dollars of Government Spending.”

Trump will take office for a second and final term on January 25, 2025.

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French newspapers sue X for using content without paying

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Major French news organisations say they are suing X, a micro-blogging platform, for allegedly using their content without paying.

According to Reuters, the newspapers, which are Le Monde, Le Figaro and Le Parisien, announced in a statement on Tuesday.

The media platforms said they were due payment under their ancillary rights, which allow payment to news outlets by digital platforms, for the distribution of their content.

They said X, formerly known as Twitter, and owned by billionaire Elon Musk, has never agreed to open negotiations with French news publishers, unlike Alphabet Inc, Google and Meta Platforms Inc.

The publication also said the media organisations claimed that X has not complied with an order issued by the Paris Court of Justice in May to release information required to calculate the amount owed.

“The revenue from these rights, with the investment that it would enable its beneficiaries to make, is a boost to the plurality, independence and quality of the media, which are essential for freedom of expression and the right to information in our democratic society,” the newspapers said.

A spokesperson for the Paris tribunal also confirmed the case to Reuters and said a hearing has been scheduled for May 15, 2025.

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