Latest news with #CASGEVY
Al Etihad
a day ago
- Health
- Al Etihad
Department of Health - Abu Dhabi highlights sickle cell awareness efforts
19 June 2025 14:30 ABU DHABI (WAM)Executive Director of the Health Life Sciences Sector at the Department of Health – Abu Dhabi (DoH), Dr. Asma Al Mannaei, affirmed that World Sickle Cell Day, marked annually on 19th June, is a vital occasion to raise awareness about the disease, support patients and their families, and shed light on efforts aimed at reducing its health and social Al Mannaei told WAM that Sickle cell disease remains a significant public health concern globally, affecting the quality of life for patients and placing long-term pressure on families. "At the Department of Health – Abu Dhabi, we are committed to reshaping that reality through innovation, early detection and personalised care. Our mission is to foster a healthy population, deliver best-in-class care, and drive continuous resilience and innovation."Dr. Al Mannaei added, "Supported by powerful Artificial Intelligence tools, driven by data and genomic sciences, we are building a system that predicts, prevents and acts.""Our efforts begin with prevention. Through the Premarital Genetic Screening Programme, we empower couples to make informed decisions that help reduce the prevalence of genetic conditions such as sickle cell in future generations."She continued, 'We then move to prediction, where we are shifting from a reactive approach to proactive and personalised care approach for sickle cell, by utilising platforms such as the Emirati Genome Programme. By integrating sickle cell disease in the newly launched Newborn Genetic Screening Programme, we can ensure early detection, timely intervention and healthier outcomes for families across the Emirate.'Dr. Al Mannaei also praised the introduction of innovative treatments. In collaboration with the Abu Dhabi Stem Cells Centre and global partner Vertex Pharmaceuticals, the Department has introduced CASGEVY, a CRISPR/Cas9-based gene-editing therapy for patients with sickle cell disease and transfusion-dependent beta-thalassaemia."This marks the first time this cutting-edge treatment is available in the UAE," she added that, beyond treatment, investment in scientific research remains a top priority. In partnership with the Authority of Social Contribution – Ma'an, and through the Research and Innovation Fund, the Department is supporting research that addresses key healthcare priorities. One of the grant awardees received funding for research focused on anaemia and sickle cell disease, examining their impact on healthspan.
Associated Press
09-05-2025
- Business
- Associated Press
Biomay Launches FDA-Grade CRISPR/Cas9 Nuclease for Off-the-Shelf Purchase
Biomay, a leading manufacturer of recombinant proteins, today announced the commercial availability of its CRISPR/Cas9 nuclease, marking a significant addition to its off-the-shelf product portfolio for genome-editing applications. Clients purchasing Biomay´s Cas9 will benefit from the company´s unparalleled track record and expertise as a market-registered GMP-manufacturer of the nuclease. Biomay is the FDA-approved manufacturer of recombinant Cas9 as the essential component of CASGEVY®, the very first CRISPR genome editing product on the market. Biomay's Cas9 (internal code 'BMC9') is based on the classical wild-type Cas9 nuclease fromStreptococcus pyogenes. The Cas9 manufacturing process has beende novodeveloped, GMP-implemented and PPQ-validated by Biomay. GMP and RUO manufacturing is performed by fermentation withE. coliand by purification with chromatographic methods. By quality control with a comprehensive set of validated analytical assays, the consistent integrity, purity and potency of the product is secured. 'The addition of Cas9 to Biomay's off-the-shelf portfolio aligns with our mission to provide high-quality and scalable solutions for emerging therapeutic modalities,'said Dr. Hans Huber, CEO of Biomay.'With this launch, we are expanding access to a critical component of gene-editing workflows, backed by our proven manufacturing expertise. Biomay´s off-the-shelf distributed Cas9, in combination with our made-to-order GMP services, will guarantee full scalability and GMP compliance throughout the whole product lifecycle. Biomay´s latest addition further strengthens the company's position as a key supplier in the field of gene and cell therapies. The CRISPR/Cas system, a transformative gene-editing technology, whose discovery was honored with the 2020 Nobel Prize in Chemistry, enables precise and efficient modification of genomic sequences. About Biomay: Biomay AG is a fully integrated Contract Development and Manufacturing Organization (CDMO) based in Vienna, Austria. Founded in 1984, the expression of recombinant proteins inE. colihas been Biomay's business focus yet from its beginning. Today, Biomay offers cGMP services for manufacturing of messenger RNA (mRNA), circular plasmid DNA, linear IVT-template DNA and therapeutic recombinant proteins. Biomay operates a dedicated mRNA Competence Center for cGMP manufacturing and QC testing of mRNA drug substance and drug product (clinical, commercial). The company's scope of services comprises process and analytical development, cell banking, R&D material supply, cGMP manufacturing, lipid nanoparticle / LNP formulation and aseptic filling. Biomay's facilities are inspected by the US FDA.
Yahoo
09-05-2025
- Business
- Yahoo
Unpacking Q1 Earnings: Vertex Pharmaceuticals (NASDAQ:VRTX) In The Context Of Other Therapeutics Stocks
The end of the earnings season is always a good time to take a step back and see who shined (and who not so much). Let's take a look at how therapeutics stocks fared in Q1, starting with Vertex Pharmaceuticals (NASDAQ:VRTX). Over the next few years, therapeutic companies, which develop a wide variety of treatments for diseases and disorders, face strong tailwinds from advancements in precision medicine (including the use of AI to improve hit rates) and growing demand for treatments targeting rare diseases. However, headwinds such as rising scrutiny over drug pricing, regulatory unknowns, and competition from larger, more resourced pharmaceutical companies could weigh on growth. The 10 therapeutics stocks we track reported a mixed Q1. As a group, revenues beat analysts' consensus estimates by 1.2%. Amidst this news, share prices of the companies have had a rough stretch. On average, they are down 10.5% since the latest earnings results. Founded in 1989 with a mission to create medicines that treat the underlying causes of disease rather than just symptoms, Vertex Pharmaceuticals (NASDAQ:VRTX) develops and markets transformative medicines for serious diseases, with a focus on cystic fibrosis, sickle cell disease, and pain management. Vertex Pharmaceuticals reported revenues of $2.77 billion, up 3% year on year. This print fell short of analysts' expectations by 2.3%. Overall, it was a disappointing quarter for the company with a significant miss of analysts' EPS estimates and full-year revenue guidance slightly missing analysts' expectations. 'Vertex delivered a strong start to 2025 with notable execution across the business as we grow and diversify the revenue base, progress multiple launches and advance the R&D pipeline. We continued to expand our leadership in CF and build global momentum for CASGEVY, and we launched JOURNAVX in moderate-to-severe acute pain,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. Unsurprisingly, the stock is down 14.1% since reporting and currently trades at $429.37. Read our full report on Vertex Pharmaceuticals here, it's free. Founded by a mother seeking treatment for her daughter's pulmonary arterial hypertension, United Therapeutics (NASDAQ:UTHR) develops and commercializes medications for chronic lung diseases and other life-threatening conditions, with a focus on pulmonary hypertension treatments. United Therapeutics reported revenues of $794.4 million, up 17.2% year on year, outperforming analysts' expectations by 5.6%. The business had an exceptional quarter with an impressive beat of analysts' EPS estimates. The market seems content with the results as the stock is up 2.7% since reporting. It currently trades at $308. Is now the time to buy United Therapeutics? Access our full analysis of the earnings results here, it's free. Founded in 1991 as one of the pioneers in translating genetic discoveries into clinical applications, Myriad Genetics (NASDAQ:MYGN) develops genetic tests that assess disease risk, guide treatment decisions, and provide insights across oncology, women's health, and mental health. Myriad Genetics reported revenues of $195.9 million, down 3.1% year on year, falling short of analysts' expectations by 2.3%. It was a softer quarter as it posted full-year revenue guidance missing analysts' expectations. As expected, the stock is down 44.7% since the results and currently trades at $4.01. Read our full analysis of Myriad Genetics's results here. Rising to global prominence during the COVID-19 pandemic with one of the first effective vaccines, Moderna (NASDAQ:MRNA) develops messenger RNA (mRNA) medicines that direct the body's cells to produce proteins with therapeutic or preventive benefits for various diseases. Moderna reported revenues of $108 million, down 35.3% year on year. This result lagged analysts' expectations by 8.4%. It was a softer quarter as it also produced full-year revenue guidance missing analysts' expectations. Moderna had the weakest performance against analyst estimates, slowest revenue growth, and weakest full-year guidance update among its peers. The stock is down 14.5% since reporting and currently trades at $24.44. Read our full, actionable report on Moderna here, it's free. Born from a 2013 spinoff of Abbott Laboratories' pharmaceutical business, AbbVie (NYSE:ABBV) is a biopharmaceutical company that develops and markets medications for autoimmune diseases, cancer, neurological disorders, and other complex health conditions. AbbVie reported revenues of $13.34 billion, up 8.4% year on year. This number surpassed analysts' expectations by 3.3%. Overall, it was a very strong quarter as it also recorded an impressive beat of analysts' constant currency revenue estimates and a decent beat of analysts' EPS estimates. The stock is up 3% since reporting and currently trades at $185.99. Read our full, actionable report on AbbVie here, it's free. Thanks to the Fed's rate hikes in 2022 and 2023, inflation has been on a steady path downward, easing back toward that 2% sweet spot. Fortunately (miraculously to some), all this tightening didn't send the economy tumbling into a recession, so here we are, cautiously celebrating a soft landing. The cherry on top? Recent rate cuts (half a point in September 2024, a quarter in November) have propped up markets, especially after Trump's November win lit a fire under major indices and sent them to all-time highs. However, there's still plenty to ponder — tariffs, corporate tax cuts, and what 2025 might hold for the economy. Want to invest in winners with rock-solid fundamentals? Check out our Hidden Gem Stocks and add them to your watchlist. These companies are poised for growth regardless of the political or macroeconomic climate. Join Paid Stock Investor Research Help us make StockStory more helpful to investors like yourself. Join our paid user research session and receive a $50 Amazon gift card for your opinions. Sign up here.
CNBC
06-05-2025
- Health
- CNBC
CRISPR-based gene editing revolutionized medicine—what's next for the firm that helped develop it?
CRISPR-Cas 9 is a gene-editing tool that made it possible to rewrite any organism's genetic code and tackle genetic diseases more effectively. Known as genetic scissors, CRISPR identifies a DNA sequence that is cut by an enzyme called Cas 9. It then changes or replaces that sequence with a different section of DNA. For this discovery, co-inventors Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize in Chemistry in 2020. "By our interest in the lab to find new molecules that could have a role in in the bacterium streptococcus pardonus , we came across a very neat mechanism that allows to really recognize the virus that infects the bacterium in a very, very specific minor at the level of the genome of the virus. And we exploited this natural mechanism to develop the CRISPR-Cas9 technology," Emmanuelle Charpentier said in an interview with CNBC's The Edge. In 2013, Charpentier co-founded CRISPR Therapeutics to fulfil her lifelong goal of finding cures for diseases. A decade later, the company and its partner Vertex Pharmaceuticals developed CASGEVY, a therapy to treat blood disorders beta thalassemia and sickle cell disease. "With CASGEVY, we're taking the bone marrow cells from the patient, making the edit for that particular patient and we're putting it back into the patient, and it reconstitutes the hematopoietic system of the patient. We're making a drug just for you," CRISPR Therapeutics' CEO Samarth Kulkarni told The Edge. CASGEVY is a one-time therapy that costs $2.2 million per patient and can be administrated on patients 12 years of age and older. In 2023, it became the first CRISPR-based gene editing therapy to be approved by the Federal Drug Administration. CRISPR Therapeutics currently has seven clinical and ten pre-clinical programs across oncology, autoimmune cardiovascular disease and diabetes, and is investigating next generation editing modalities. Watch the video above for the full interview with Professor Charpentier from Berlin, Germany, and a tour of CRISPR Therapeutics' facilities in Boston, Massachusetts.
Yahoo
06-05-2025
- Business
- Yahoo
Why Did Vertex Pharmaceuticals Stock Fall On Tuesday?
On Monday, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) reported lower-than-expected first-quarter 2025 earnings. Vertex reported first-quarter adjusted earnings per share of $4.06, down from $4.76 a year ago, missing the consensus of $4.32. The cystic fibrosis-focused company reported sales of $2.77 billion, missing the consensus of $2.85 billion. Total revenue increased by 3%, primarily driven by the continued performance of Trikafta/Kaftrio and an early contribution from the U.S. launch of Alyftrek. In the U.S., total revenue increased 9% to $1.66 billion due to continued strong patient demand and higher net realized pricing. Outside the U.S., total revenue decreased 5% to $1.11 billion, as strong patient demand in established and newer markets was offset by the expected revenue decline in Russia, where Vertex is experiencing a violation of its intellectual property rights. Also Read: Vertex Discontinues Development Of Type 1 Diabetes Candidate After Disappointing Data 'Vertex delivered a strong start to 2025 with notable execution across the business as we grow and diversify the revenue base, progress multiple launches and advance the R&D pipeline. We continued to expand our leadership in CF and build global momentum for CASGEVY, and we launched JOURNAVX in moderate-to-severe acute pain,' said Reshma Kewalramani, CEO and President of Vertex. 'With multiple programs in pivotal development including povetacicept, which continues to make rapid progress in achieving its potential as a pipeline-in-a-product, and additional programs in early and mid-stage development, Vertex is poised to continue to deliver value for years to come.' Guidance: Vertex raised the low end of total revenue guidance by $100 million to $11.85 billion-$12 billion from $11.75 billion-$12 billion, compared to the consensus of $11.97 billion. The guidance also includes an immaterial cost impact from tariffs in 2025 based on currently known tariff rates and regulations. Vertex has temporarily paused the multiple ascending dose portion of the Phase 1/2 study of VX-522, a nebulized CFTR mRNA therapy, to assess a tolerability issue. William Blair writes, 'While the pause is unfortunate, we believe that the decision to pause the study rather than discontinue it suggests that, beyond the tolerability issue, management is encouraged by the data generated to date given it is open-label.' Analyst Myles Minter writes, 'While there was a slight miss on the top line leading to shares trading off 3% in the aftermarket, management raised the lower end of total revenue guidance, suggesting Street estimates may not have fully encompassed the previously disclosed impacts of an unauthorized Trikafta copy in Russia. We would be buyers on weakness here as the CF franchise still represents one of the most stable long-term growth franchises with a competitive moat in the biotech sector.'
