RARE 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The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.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 This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of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).

The major averages had a mixed finish as semiconductor stocks pulled back following a massive multi-week run. AI-driven enthusiasm pushed the S&P 500 and Nasdaq to fresh records yesterday, but investors are now confronting a harder macro backdrop than previously anticipated. Meanwhile, crude is back above $100 as hopes for a durable U.S.-Iran ceasefire continue to deteriorate, with President Trump describing negotiations as being "on life support." Of note, the Iranian Fars news service reportedly said that Iran will not join the next round of U.S. talks without five preconditions being met.Get caught up quickly on the top news and calls moving stocks with these five Top Five lists.1. STOCK NEWS:eBay'sboard rejected GameStop'sUnder Armourreported mostly in-line Q4 results andActivist investor Nelson Peltz's Trian Fund Management is seeking investor backing for a bid to take Wendy'sprivate,BuzzFeedannounced a proposed majority investment byFDA Commissioner Marty Makary has, according to multiple media reports citing administration officials2. WALL STREET CALLS:Lowe'sto Buy at CitiAutodeskat Buy from Neutral at BofARothschildToaston risk from DoorDashU.S. rolloutZoomInfoto Underweight at Piper Sandler and to Neutral-equivalent ratings and several other firmsWall Streetof drone-maker Aevexafter its recent IPO3. AROUND THE WEB:Microsofthas already earned back more than double the capital it invested in OpenAI in terms of new revenue from services related to OpenAI, The Information reportsGoogleand Elon Musk's SpaceX are in talks for a rocket launch deal amid efforts to put orbital data centers in space, WSJ saysAlphabet'sYouTube is looking to connect creators with sponsors to secure funding for shows before they launch, NY Times reportsSoftBankis partnering with South Korea's Cosmos Lab and DeltaX to mass produce next-gen data center batteries in Sakai, Japan at a scale of 1 GWh/year from FY27, Bloomberg reportsBoeing'ssuccess hinges on its successor to the 737, Bloomberg says4. MOVERS:Zebra Technologiesrose after providing areport for Q1SI-Bonewas higher after also providing areport for Q1Gitlabfell after announcing aZoomInfowas lower afterand reiterating its FY26 outlookGoProdeclined after reportingand withdrawing its FY26 guidance5. EARNINGS/GUIDANCE:Sea LimitedreportedBayerprovided areport for Q1Qnity Electronicsalso provided areport for Q1Tencent Musicreported ain Q1 earningsHims & Hers Healthprovidedbut raised its FY26 revenue guidanceINDEXES:The Dow rose 56.09, or 0.11%, to 49,760.56, the Nasdaq lost 185.92, or 0.71%, to 26,088.20, and the S&P 500 declined 11.88, or 0.16%, to 7,400.96.