<p>The US Food and Drug Administration (FDA) has given the green light to two new gene therapies for sickle cell disease.&nbsp;One of them, called Casgevy, is a treatment that uses the CRISPR gene editing technology, which won the Nobel Prize. The other approved therapy is Lyfgenia, developed by bluebird bio. Both treatments are meant for individuals aged 12 and above.</p>

<p>Casgevy is developed by Vertex Pharmaceuticals and CRISPR Therapeutics and comes with a price tag of $2.2 million, while bluebird's Lyfgenia is priced at $3.1 million. These are considered one-time treatments and are expected to be available in early 2024, media reports said.&nbsp;</p>

<p>Sickle Cell Disease (SCD) is an inherited blood disorder that causes pain and complications due to the production of flawed, sickle-shaped hemoglobin. This impairs the proper transport of oxygen by red blood cells to the body's tissues. It affects around 100,000 people in the United States, primarily among the Black population.&nbsp;</p>

<p>Also Read |&nbsp;<a href="https://www.wionews.com/science/researchers-develop-vr-goggles-for-mice-to-create-immersive-scenarios-for-brain-research-667802">Researchers develop VR goggles for mice to create immersive scenarios for brain research</a></p>

<p>The approval is a crucial advancement for the sickle cell disease community, which according to Robert Brodsky, President of the American Society of Hematology, has historically faced neglect and underfunding.&nbsp;</p>

<p>Also Read |&nbsp;<a href="https://www.wionews.com/science/penguins-sleep-for-few-seconds-at-a-time-over-24-hours-to-protect-eggs-study-667666">Penguins sleep for few seconds at a time over 24 hours to protect eggs: Study</a></p>

<p>In clinical trials, both gene therapies demonstrated effectiveness in reducing painful episodes in patients with sickle cell disease. Casgevy, based on CRISPR technology, uses molecular "scissors" to correct faulty genes. On the other hand, Lyfgenia works by introducing modified genes into the body through disabled viruses.</p>

<h2><strong>Cancer risks?&nbsp;</strong></h2>

<p>The FDA has issued a warning on Lyfgenia's label regarding the risk of blood cancer after two patients in bluebird's trial developed acute myeloid leukemia. The FDA will closely monitor potential side effects.</p>

<p><strong>Watch |&nbsp;<a href="https://www.wionews.com/videos/cop28-indigenous-activists-dance-to-deliver-message-in-dubai-667846">COP28: Indigenous activists dance to deliver message in Dubai</a></strong></p>

<div class="ui-state-default" contenteditable="false">
<div class="twitter-block" draggable="false">
<div class="videoWrapper trancodeVideo"><iframe allowfullscreen="" autoplay="1" class="includeVideo" frameborder="0" height="350" src="https://www.wionews.com/videos/cop28-indigenous-activists-dance-to-deliver-message-in-dubai-667846/embed" style="width:100%;max-width:560px;" width="560"></iframe></div>
</div>
<a class="close-mark close-div" href="#" id="jqclosemark">×</a></div>

<p>While both therapies show promise, data on their long-term effects is limited. The companies plan to assess potential long-term safety risks through a 15-year follow-up study after approval. Patients will be monitored throughout their lives for cancer malignancies.</p>

<p><em>(With inputs from agencies)</em></p>


The US Food and Drug Administration (FDA) has given the green light to two new gene therapies for sickle cell disease. One of them, called Casgevy, is a treatment that uses the CRISPR gene editing technology, which won the Nobel Prize.

The US Food and Drug Administration (FDA) has given the green light to two new gene therapies for sickle cell disease. One of them, called Casgevy, is a treatment that uses the CRISPR gene editing technology, which won the Nobel Prize. The other approved therapy is Lyfgenia, developed by bluebird bio. Both treatments are meant for individuals aged 12 and above. Casgevy is developed by Vertex Pharmaceuticals and CRISPR Therapeutics and comes with a price tag of $2.2 million, while bluebird's Lyfgenia is priced at $3.1 million. These are considered one-time treatments and are expected to be available in early 2024, media reports said. Sickle Cell Disease (SCD) is an inherited blood disorder that causes pain and complications due to the production of flawed, sickle-shaped hemoglobin. This impairs the proper transport of oxygen by red blood cells to the body's tissues. It affects around 100,000 people in the United States, primarily among the Black population. Also Read | Researchers develop VR goggles for mice to create immersive scenarios for brain research The approval is a crucial advancement for the sickle cell disease community, which according to Robert Brodsky, President of the American Society of Hematology, has historically faced neglect and underfunding. Also Read | Penguins sleep for few seconds at a time over 24 hours to protect eggs: Study In clinical trials, both gene therapies demonstrated effectiveness in reducing painful episodes in patients with sickle cell disease. Casgevy, based on CRISPR technology, uses molecular "scissors" to correct faulty genes. On the other hand, Lyfgenia works by introducing modified genes into the body through disabled viruses. Cancer risks? The FDA has issued a warning on Lyfgenia's label regarding the risk of blood cancer after two patients in bluebird's trial developed acute myeloid leukemia. The FDA will closely monitor potential side effects. Watch | COP28: Indigenous activists dance to deliver message in Dubai × While both therapies show promise, data on their long-term effects is limited. The companies plan to assess potential long-term safety risks through a 15-year follow-up study after approval. Patients will be monitored throughout their lives for cancer malignancies. (With inputs from agencies)

New hope for Sickle Cell Anaemia patients as FDA gives nod to Casgevy, Lyfgenia gene therapies

<p>India will launch&nbsp;an ambitious nationwide mission to eliminate sickle cell anaemia by 2047, country's finance minister Nirmala Sitharaman announced in the annual budget of world's fifth largest economy on February 1. Sickle cell anaemia, a genetic disease with high fatality rate&nbsp;that alters the shape of blood cells bringing down their capacity to carry oxygen in the body, is a widespread genetic condition in Central India's areas with significant tribal population.&nbsp;</p>

<p><strong>India's mission against sickle cell anaemia: What does it entail?</strong></p>

<p>India's finance minister Nirmala Sitharaman told parliament in New Delhi that the mission will be a collaborative effort between federal government and individual state administrations.</p>

<p><strong>ALSO READ |&nbsp;<a href="https://www.wionews.com/business-economy/budget-2023-live-updates-nirmala-sitharaman-to-present-union-budget-2023-24-in-parliament-today-557696">India Union Budget 2023: Growth at 7 pc in current fiscal, highest among major economies: Nirmala Sitharaman</a></strong></p>

<p>"It will entail awareness creation, universal screening of 7 crore (70 million) people in the age-group of 0 to 40 years in affected tribal areas and counseling through collaborative efforts of central ministries and state governments," Sitharaman said.</p>

<p>According to India's Ministry of Tribal Affairs, about 1 in 86 children in tribal population are born with sickle Cell Anaemia condition. &nbsp;</p>

<p>Experts say that early detection and treatment of SDC is of "utmost importance for improved health conditions as a nation".&nbsp;</p>

<p>In Budget 2023, India's finance minister, among other initiatives for improving country's health infrastructure, announced that as many as 157 new nursing colleges will be established in the core locations of the existing 157 medical colleges established since 2014.</p>

<p><strong>ALSO WATCH |&nbsp;<a href="https://www.wionews.com/videos/union-budget-2023-will-government-spend-support-growth-or-populism-557747">Union Budget 2023: Will government spend support growth or populism?</a></strong></p>

<div class="ui-state-default" contenteditable="false">
<div class="twitter-block" draggable="false">
<div class="videoWrapper trancodeVideo"><iframe allowfullscreen="" autoplay="1" class="includeVideo" frameborder="0" height="350" src="https://www.wionews.com/videos/union-budget-2023-will-government-spend-support-growth-or-populism-557747/embed" style="width:100%;max-width:560px;" width="560"></iframe></div>
</div>
<a class="close-mark close-div" href="#" id="jqclosemark">×</a></div>

<p>"Facilities of Indian Council of Medical Research (ICMR) labs will be made available for private R&amp;D (research and development) teams to encourage collaborative work," she added.&nbsp;</p>

<p><strong>WATCH WION LIVE HERE</strong></p>

<p><iframe allow="accelerometer; autoplay; encrypted-media; gyroscope; picture-in-picture" allowfullscreen="" frameborder="0" height="350" loading="”lazy”" src="https://www.wionews.com/live-tv/embed?autoplay=1&amp;mute=1  width=&quot;100%&quot;"></iframe></p>

<p>You can <strong>now write for wionews.com</strong> and be a part of the community. Share your stories and opinions with us <strong><a href="https://www.wionews.com/content-contribution-registration-form">here</a>.</strong></p>


India will launch an ambitious nationwide mission to eliminate sickle cell anaemia by 2047, country's finance minister Nirmala Sitharaman announced in the annual budget of world's fifth largest economy on February 1.

India will launch an ambitious nationwide mission to eliminate sickle cell anaemia by 2047, country's finance minister Nirmala Sitharaman announced in the annual budget of world's fifth largest economy on February 1. Sickle cell anaemia, a genetic disease with high fatality rate that alters the shape of blood cells bringing down their capacity to carry oxygen in the body, is a widespread genetic condition in Central India's areas with significant tribal population. India's mission against sickle cell anaemia: What does it entail? India's finance minister Nirmala Sitharaman told parliament in New Delhi that the mission will be a collaborative effort between federal government and individual state administrations. ALSO READ | India Union Budget 2023: Growth at 7 pc in current fiscal, highest among major economies: Nirmala Sitharaman "It will entail awareness creation, universal screening of 7 crore (70 million) people in the age-group of 0 to 40 years in affected tribal areas and counseling through collaborative efforts of central ministries and state governments," Sitharaman said. According to India's Ministry of Tribal Affairs, about 1 in 86 children in tribal population are born with sickle Cell Anaemia condition. Experts say that early detection and treatment of SDC is of "utmost importance for improved health conditions as a nation". In Budget 2023, India's finance minister, among other initiatives for improving country's health infrastructure, announced that as many as 157 new nursing colleges will be established in the core locations of the existing 157 medical colleges established since 2014. ALSO WATCH | Union Budget 2023: Will government spend support growth or populism? × "Facilities of Indian Council of Medical Research (ICMR) labs will be made available for private R&D (research and development) teams to encourage collaborative work," she added. WATCH WION LIVE HERE You can now write for wionews.com and be a part of the community. Share your stories and opinions with us here.

India Budget 2023: Govt announces mission to eliminate sickle cell anaemia by 2047

sickle-cell-anaemia

Sickle Cell Anaemia, News on Sickle Cell Anaemia, stories on Sickle Cell Anaemia, articles on Sickle Cell Anaemia, Sickle Cell Anaemia stories at Wion, Sickle Cell Anaemia news, Sickle Cell Anaemia latest news

sickle cell-anaemia

New hope for Sickle Cell Anaemia patients as FDA gives nod to Casgevy, Lyfgenia gene therapies

India Budget 2023: Govt announces mission to eliminate sickle cell anaemia by 2047