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The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.
This is particularly the case for pediatric forms of epilepsy where geneediting techniques like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are becoming more important, says Ferraro. GW Pharmaceuticals’ Epidiolex (cannabidiol), the first-FDAapproved CBD treatment , is also a valuable addition, he adds.
New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.
In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. At the moment, the only FDA-approved treatments for ATTR cardiomyopathy are Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), which were given a green light by the agency two years ago.
Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.
Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. Additional research is underway using geneediting via CRISPR, primarily investigating genes with known relationships to ALS, such as SOD1 and C9orf72.
The HELIOS-B results build on earlier success from the HELIOS-A trial, which led to the US Food and Drug Administration (FDA) approval of vutrisiran (Amvuttra) for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.
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.
Paxlovid was first authorized under the FDA Emergency Use Authorization in December 2021 ; however, it has received FDAapproval 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 FDAapproval.
The RNA Revolution: From mRNA Vaccines to RNA Editing. RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech.
GeneEditing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, geneediting can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.
Clinical interest in bsAbs gained momentum following the promising data and market success of blinatumomab (BLINCYTO®, Amgen), a fragment-based bispecific T cell engager (BiTE), which first received FDAapproval in 2014 and EMA approval in 2015.
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 GeneEditing Inventors Win Nobel Prize.
Casgevy: A Game-Changer for Beta Thalassemia CRISPR Therapeutics and Vertex are transforming blood disorder treatment with their gene-editing solution Casgevy (exagamglogene autotemcel [exa-cel]). This one-time gene therapy tackles transfusion-dependent beta-thalassemia (TDT), a disorder causing severe anemia.
RNA-Based Therapeutics RNA molecules have emerged as promising therapeutic agents due to their ability to precisely target undruggable proteins or molecules, often with minimal side effects. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.
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