DSGN News & Events

Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas pledged to prevent political interference, Endpoint News' Zachary Brennan reports. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells. "Today's action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research. "By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on.Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible." This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

Stocks finished mixed to start the week as investors continue to watch climbing oil prices while awaiting more developments out of the Middle East. Investors are increasingly worried that inflation is becoming entrenched due to energy prices, persistent fiscal deficits and resilient economic demand. Markets are now pricing in a meaningful possibility that the Federal Reserve could raise rates again later this year instead of cutting them.Of note, President Trump stated in athat he was asked by the leaders of several Middle Eastern nations to hold off a planned military attack on Iran, which was scheduled for tomorrow, but added that he has instructed the U.S. military leadership "to be prepared to go forward with a full, large scale assault of Iran, on a moment's notice, in the event that an acceptable Deal is not reached."Get caught up quickly on the top news and calls moving stocks with these five Top Five lists.1. STOCK NEWS:Dominionand NextEra Energyin an all-stock transactionPublicisLiveRampfor $2.2BFordand EDF Group announced the signing of aTrump plans to announce a "dramatic expansion" of discounted prescription medication offerings available on TrumpRx,A U.S. jury on Monday ruled against Elon Musk in his lawsuit against OpenAI, finding the AI company not liable to the Teslafounder for having allegedly strayed from its original mission to benefit humanity,2. WALL STREET CALLS:F5to Outperform from In Line at Evercore ISIZscalerto Buy at B. RileyCoreWeaveand Nebiuswith lowered Neutral ratings as a new analyst took over coverage at DA DavidsonApplied Materialsto Equal Weight at Morgan StanleySalesforcewith an Underperform at BofA amid structural reset3. AROUND THE WEB:NvidiaCEO Jensen Huang told Bloomberg that it is up to China to decide on AI chips and that he looks forward to China being a more open marketTakedahas been ordered to pay hundreds of millions for violating antitrust law by delaying the generic version of its branded Amitiza drug, Bloomberg saysApple'srevamped Siri may launch in beta, and will have an option to auto-delete chats, Bloomberg reportsPresident Trump said he "should have asked for more" of a stake in Intelon behalf of the U.S. government, Fortune saysVinFast'splanned sale of two Vietnamese factories will allow the company to shed $6.9B in debt and obligations to help assist its path to profitability, Bloomberg says4. MOVERS:Hive Digitalwas higher afteris planning to build a new AI infrastructure facility in the Greater Toronto Area with approximately 320 megawatts of utility capacityCourseraincreased after announcing aServiceNowgained after BofAcoverage of the stock with a Buy ratingDesign Therapeuticswas lower afterRegeneronfell after its Phase 3 trial of fianlimab5. EARNINGS/GUIDANCE:Bradyand raised its guidance for FY26iQiyi, with EPS and revenue lower year-over-yearGossamer Bio, with CEO Faheem Hasnain commenting, "We are executing across multiple fronts at Gossamer"RedCloudreportedand reaffirmed guidance for FY26Baidu, with EPS and revenue lower year-over-yearINDEXES:The Dow rose 159.95, or 0.32%, to 49,686.12, the Nasdaq lost 134.41, or 0.51%, to 26,090.73, and the S&P 500 declined 5.45, or 0.074%, to 7,403.05.

Stocks were mostly lower near noon following a record-setting week as investors continue to watch climbing oil prices while awaiting more developments out of the Middle East. The 30-year Treasury yield climbed above 5.15%, its highest level since before the 2008 financial crisis, while the 10-year Treasury yield rose toward 4.65%. Investors are increasingly worried that inflation is becoming entrenched due to energy prices, persistent fiscal deficits and resilient economic demand. Markets are now pricing in a meaningful possibility that the Federal Reserve could raise rates again later this year instead of cutting them.Semiconductor and mega-cap tech stocks remain the market's leadership group, but investors are becoming more sensitive to valuations as borrowing costs rise. Analysts are increasingly debating whether AI enthusiasm has become excessive, particularly given the massive capital spending required to sustain the boom.Get caught up quickly on the top news and calls moving stocks with these five Top Five lists.1. STOCK NEWS:Dominionand NextEra Energyin an all-stock transactionPublicisLiveRampfor $2.2BFordand EDF Group announced the signing of aRegeneronsaid its Phase 3 trial of fianlimabMacy'sand Delta Air Lineswere higher after Berkshire Hathaway2. WALL STREET CALLS:F5to Outperform from In Line at Evercore ISIZscalerto Buy at B. RileyCoreWeaveand Nebiuswith lowered Neutral ratings as a new analyst took over coverage at DA DavidsonApplied Materialsto Equal Weight at Morgan StanleySalesforcewith an Underperform at BofA amid structural reset3. AROUND THE WEB:Apple'srevamped Siri may launch in beta, and will have an option to auto-delete chats, Bloomberg reportsU.S. President Donald Trump said he "should have asked for more" of a stake in Intelon behalf of the U.S. government, Fortune saysFugazi Research issued a cautious note on MMTECActivist investor Elliot Investment Management has built a big stake in Bio-Rade Laboratories, with plans to push the company to boost its underperforming stock price, WSJ saysVinFast'splanned sale of two Vietnamese factories will allow the company to shed $6.9B in debt and obligations to help assist its path to profitability, Bloomberg says4. MOVERS:Hive Digitalhigher after announcing its subsidiary is planning to build a newin the Greater Toronto Area with approximately 320 megawatts of utility capacityCourseraincreases after announcing a $500MServiceNowgains after BofAof the stock with a Buy ratingDesign Therapeuticslower afterfrom its RESTORE-FA trialPoet Technologiesfalls after closing a5. EARNINGS/GUIDANCE:Bradyand raised its guidance for FY26iQiyi, with EPS and revenue lower year-over-yearGossamer Bio, with CEO Faheem Hasnain commenting, "We are executing across multiple fronts at Gossamer"RedCloudand reaffirmed guidance for FY26Baidu, with EPS and revenue lower year-over-yearINDEXES:Near midday, the Dow was up 0.02%, or 12.01, to 49,538.18, the Nasdaq was down 0.60%, or 157.04, to 26,068.11, and the S&P 500 was down 0.25%, or 18.29, to 7,390.21.