Latest news with #CASGEVY
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.'
Yahoo
05-05-2025
- Business
- Yahoo
Vertex Pharmaceuticals (NASDAQ:VRTX) Reports Sales Below Analyst Estimates In Q1 Earnings
Biotech company Vertex Pharmaceuticals (NASDAQ:VRTX) missed Wall Street's revenue expectations in Q1 CY2025 as sales rose 3% year on year to $2.77 billion. The company's full-year revenue guidance of $11.88 billion at the midpoint came in 0.9% below analysts' estimates. Its non-GAAP profit of $4.06 per share was 5.4% below analysts' consensus estimates. Is now the time to buy Vertex Pharmaceuticals? Find out in our full research report. Revenue: $2.77 billion vs analyst estimates of $2.83 billion (3% year-on-year growth, 2.3% miss) Adjusted EPS: $4.06 vs analyst expectations of $4.29 (5.4% miss) Adjusted Operating Income: $1.18 billion vs analyst estimates of $1.30 billion (42.7% margin, 9.1% miss) The company reconfirmed its revenue guidance for the full year of $11.88 billion at the midpoint Operating Margin: 22.7%, down from 42.4% in the same quarter last year Market Capitalization: $128.8 billion '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. 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. A company's long-term sales performance is one signal of its overall quality. Any business can experience short-term success, but top-performing ones enjoy sustained growth for years. Over the last five years, Vertex Pharmaceuticals grew its sales at an impressive 18.2% compounded annual growth rate. Its growth beat the average healthcare company and shows its offerings resonate with customers. We at StockStory place the most emphasis on long-term growth, but within healthcare, a half-decade historical view may miss recent innovations or disruptive industry trends. Vertex Pharmaceuticals's annualized revenue growth of 9.8% over the last two years is below its five-year trend, but we still think the results were respectable. This quarter, Vertex Pharmaceuticals's revenue grew by 3% year on year to $2.77 billion, falling short of Wall Street's estimates. Looking ahead, sell-side analysts expect revenue to grow 9.2% over the next 12 months, similar to its two-year rate. This projection is particularly noteworthy for a company of its scale and suggests the market sees success for its products and services. Unless you've been living under a rock, it should be obvious by now that generative AI is going to have a huge impact on how large corporations do business. While Nvidia and AMD are trading close to all-time highs, we prefer a lesser-known (but still profitable) stock benefiting from the rise of AI. Click here to access our free report one of our favorites growth stories. Adjusted operating margin is an important measure of profitability as it shows the portion of revenue left after accounting for all core expenses – everything from the cost of goods sold to advertising and wages. It's also useful for comparing profitability across companies because it excludes non-recurring expenses, interest on debt, and taxes. Vertex Pharmaceuticals has been a well-oiled machine over the last five years. It demonstrated elite profitability for a healthcare business, boasting an average adjusted operating margin of 40.1%. Looking at the trend in its profitability, Vertex Pharmaceuticals's adjusted operating margin decreased by 51.5 percentage points over the last five years. The company's two-year trajectory also shows it failed to get its profitability back to the peak as its margin fell by 44.3 percentage points. This performance was poor no matter how you look at it - it shows its expenses were rising and it couldn't pass those costs onto its customers. This quarter, Vertex Pharmaceuticals generated an adjusted operating profit margin of 42.7%, down 7 percentage points year on year. This contraction shows it was less efficient because its expenses grew faster than its revenue. We track the long-term change in earnings per share (EPS) for the same reason as long-term revenue growth. Compared to revenue, however, EPS highlights whether a company's growth is profitable. Sadly for Vertex Pharmaceuticals, its EPS declined by 15.6% annually over the last five years while its revenue grew by 18.2%. This tells us the company became less profitable on a per-share basis as it expanded due to non-fundamental factors such as interest expenses and taxes. We can take a deeper look into Vertex Pharmaceuticals's earnings to better understand the drivers of its performance. As we mentioned earlier, Vertex Pharmaceuticals's adjusted operating margin declined by 51.5 percentage points over the last five years. This was the most relevant factor (aside from the revenue impact) behind its lower earnings; taxes and interest expenses can also affect EPS but don't tell us as much about a company's fundamentals. In Q1, Vertex Pharmaceuticals reported EPS at $4.06, down from $4.76 in the same quarter last year. This print missed analysts' estimates. Over the next 12 months, Wall Street is optimistic. Analysts forecast Vertex Pharmaceuticals's full-year EPS of negative $0.41 will flip to positive $18.55. We struggled to find many positives in these results as its revenue, EPS, and adjusted operating income fell short of Wall Street's estimates. Overall, this quarter could have been better. The stock traded down 2.6% to $487 immediately after reporting. The latest quarter from Vertex Pharmaceuticals's wasn't that good. One earnings report doesn't define a company's quality, though, so let's explore whether the stock is a buy at the current price. The latest quarter does matter, but not nearly as much as longer-term fundamentals and valuation, when deciding if the stock is a buy. We cover that in our actionable full research report which you can read here, it's free. Sign in to access your portfolio
