Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
![[Comment] Aldosterone Synthase Inhibition in Resistant Hypertension: Promises and Unknowns](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/0a5065acfb3787a2e2e6999bae6248e9.webp)
Resistant hypertension affects up to 20 % of hypertensive patients and carries heightened cardiovascular risk. Recent phase‑3 studies of aldosterone synthase inhibitors such as baxdrostat and lorundrostat have demonstrated significant ambulatory blood‑pressure reductions, positioning them as potential fourth‑line agents beyond traditional mineralocorticoid antagonists. The commentary highlights the therapeutic promise of directly curbing aldosterone synthesis while noting gaps in long‑term safety, renal outcomes, and optimal placement within guideline algorithms. These uncertainties temper enthusiasm until larger outcome trials are completed.
Vinay Prasad, the FDA’s controversial biologics chief, has announced his departure, marking his second exit within a year. His resignation follows a public dispute over the agency’s handling of rare disease drug approvals, intensifying scrutiny of FDA decision‑making. Critics are...
A large observational study of 98,261 U.S. veterans with type 2 diabetes found that using GLP‑1 receptor agonists together with six to eight healthy lifestyle habits lowered major adverse cardiovascular events (MACE) by 43% compared with low‑habit, non‑GLP‑1 users. Both the...
Vinay Prasad, the controversial head of the FDA's Center for Biologics Evaluation and Research, will leave the agency in April after a tumultuous year marked by stricter vaccine guidelines and a new pathway for ultra‑rare gene therapies. His tenure saw...
Norgine announced a £23 million injection to expand its Hengoed, Wales, manufacturing site, taking total investment at the location to more than £50 million since 2022. The upgrade will add high‑speed, energy‑efficient production lines and increase warehousing capacity, allowing the company to...
SpyGlass Pharma completed an IPO that raised $172.5 million, selling 10.78 million shares at $16 each and listing on Nasdaq under the ticker SGP. The company’s SpyGlass Platform uses small implants to deliver drugs inside the eye, with its lead BIM‑IOL system...
Eli Lilly launched an Employer Connect platform on March 5, linking more than 15 independent program administrators with a nationwide pharmacy and telehealth network to broaden discounted access to its obesity drugs, notably the GLP‑1 Zepbound (tirzepatide) pen. The service targets employer‑sponsored...
Peptide therapies have moved from niche compounding to mainstream outpatient care, driven by telehealth platforms and rapid market growth. This expansion has attracted regulatory attention, with state boards and courts increasingly referencing USP and as compliance benchmarks. Non‑sterile peptide...
In February 2026 the FDA approved a suite of targeted therapies, including the HER2‑mutant NSCLC kinase inhibitor zognertinib and the first all‑oral acalabrutinib‑venetoclax combo for CLL/SLL, as well as a BRAF‑targeted encorafenib regimen for metastatic colorectal cancer. A portable tumor‑treating‑fields...
A first‑in‑human phase 1 trial at UC Davis evaluated placental mesenchymal stem cells delivered intra‑uterinely to fetuses with myelomeningocele. Six pregnancies treated between June 2021 and December 2022 resulted in intact repair sites, no cerebrospinal fluid leaks, infections, or tumor formation, and MRI scans...
Geopolitical tensions between the U.S., Israel and Iran have rattled traditional safe‑haven assets, with gold and consumer staples slipping as oil prices breach $100 per barrel. UBS recommends pharmaceutical stocks as the new defensive refuge, citing their inverse link to...
EpiciphAI, a Chinese pre‑seed biotech, is building a liquid biopsy that reads histone modifications on cell‑free chromatin to pinpoint a fragment’s tissue of origin and its disease state. Unlike most epigenetic tests that focus on DNA methylation, this platform targets...
Gee, who could this "senior FDA official" be? It's a head scratcher. But he might tell Vinay Prasad that thinly veiled attacks against, oh, UniQure, and going off on advisory councils is a great way of keeping the political pot...
Canada’s Public Health Agency has signed a new agreement with CSL Seqirus to provide millions of doses of adjuvanted cell‑based influenza vaccine in the event of a WHO‑declared pandemic. The contract replaces a previous egg‑based arrangement and builds on the...
The FDA staged a media call where an anonymous senior official publicly criticized UniQure’s experimental Huntington’s disease gene therapy. The official, identified only as a practicing hematology‑oncology professor, framed his comments as serving the public interest while hinting at personal...
At AAAAI 2026, Novartis presented data confirming remibrutinib’s rapid and sustained efficacy in chronic spontaneous urticaria, with a 25 mg BID dose delivering significant UAS7 reductions. Phase 2 trials also demonstrated the BTK inhibitor’s potential in peanut allergy, achieving 86.7% tolerance at...
The FDA’s Drug Shortages portal aggregates letters, temporary import authorizations, and annual reports that detail current and historic medication scarcities. Recent communications include temporary import permits for Lederle Leucovorin, Tafenoquine, and Physostigmine to bridge supply gaps. The site also hosts...
The article outlines the legal requirement that U.S. wholesale drug distributors and third‑party logistics providers (3PLs) must hold a valid state license to handle prescription medications. It warns that unlicensed or unauthorized partners can expose patients to counterfeit, stolen, or...
Medochemie, Cyprus’s largest generic drug maker, has powered all nine of its GMP‑certified manufacturing sites with 100% renewable electricity as of 1 October 2025, covering roughly 18 million kWh annually and eliminating thousands of tonnes of CO₂. The transition aligns the firm with tightening...
Johnson & Johnson’s Tecvayli and Darzalex combination received FDA approval for second‑line multiple myeloma treatment, marking the third drug cleared under the Commissioner’s National Priority Voucher (CNPV) program. The decision was rendered in just 55 days after J&J’s filing, thanks...
The FDA has issued a draft Level 1 guidance (Docket FDA‑2025‑D‑1504) titled “Responding to FDA Form 483 Observations at the Conclusion of a Drug CGMP Inspection.” The document targets both foreign and domestic manufacturers of human and animal drugs regulated by CDER,...
The March 3, 2026 AJMC/MHE webinar examined the Trump administration’s Great Healthcare Plan, focusing on most‑favored‑nation (MFN) drug pricing, the lapse of ACA marketplace subsidies, and the Inflation Reduction Act’s (IRA) impact on community oncology. Sixteen manufacturers have voluntarily adopted MFN pricing,...
Three biopharma firms—Bavarian Nordic, Sarepta Therapeutics, and Alkermes—announced the departures of long‑standing CEOs, prompting short‑term share declines. Bavarian Nordic’s Paul Chaplin steps down after a blocked $3 billion buyout, while Sarepta’s Doug Ingram leaves amid family health issues and recent product...
Especially after the recent disappointing regulatory update regarding $QURE AMT-130 clinical candidate for treating Huntington disease and the FDA refusal to move forward 🧵👇- here is an excellent Infograph by the BBC which explains how exactly the mechanism of @uniQure_NV’s...
Human Growth Hormone May Be Detrimental When Used to Accelerate Recovery from Acute Tendon-Bone Interface Injuries https://t.co/L4EOFMKjys
Immuneering Corporation announced its 2025 financial results, highlighting a 64% overall survival rate at 12 months for atebimetinib plus modified gemcitabine/nab‑paclitaxel in first‑line pancreatic cancer. The company secured FDA and EMA alignment on the design of its pivotal Phase 3 MAPKeeper 301...
Assuming this is real it may have something to do with their “must pay by Venmo to the following name” (at least that’s how it used to be). FDA is easing up on some aspects of peptides but gray market...
PEP protects against age-related inflammation, puts the brake on the aging process in mice, high levels in humans correlate with low inflammation So what is PEP, you ask? phosphoenolpyruvate, from glucose metabolism, a, master regulator, blocks cGAS-STING https://t.co/glMvaHSjOq https://t.co/MSVMBJbHm4
Insilico Medicine and TaiGen announced that enrollment for the Phase‑I trial of ISM4808, an AI‑designed therapy for CKD‑related anemia, is now complete and the first patient has been dosed. The study comprises single‑ascending‑dose and multiple‑ascending‑dose cohorts evaluating safety and pharmacokinetics...
Roche, Zealand shares fall on ‘undifferentiated’ obesity drug results https://t.co/5pSR2vcHBD by @Lilah_Alvarado $RHHBY $ZEAL $LLY $NVO #obesity
Servier to build cancer drug pipeline with $2.5B purchase of Day One https://t.co/aH0z8jt9tr by @realJacobBell $DAWN #biotech
Private equity firms are actively pursuing growth in the active pharmaceutical ingredient (API) sector, with five recent transactions highlighted by Astorg, EQT, New Mountain Capital and TA Associates. The deals involve both acquisitions and divestitures of API manufacturing assets, reflecting...
New from me on $QURE and FDA. Free, no paywall. The FDA, urged to avoid controversy, creates a new headache with attack against UniQure Anonymous diatribe from a senior official plunges agency back into headlines https://t.co/ceDE0TXjdI
HS-20093, a B7-H3-targeted antibody-drug conjugate in lung cancer: Results from the ARTEMIS-001 phase 1a/b trial https://t.co/Jr90iovVeo https://t.co/Nl2WkprjU3
Researchers have engineered a magnetic nanocarrier (IO@MBD) that combines γ‑Fe₂O₃ nanoparticles with a melamine‑based dendrimer to deliver doxorubicin. The platform achieves roughly 17 wt% drug loading, remains dispersible in water, and releases the drug preferentially under acidic conditions typical of tumor...
The edge is always weirder and more surprising than we can imagine, excited to follow the work of these 14 scientists and innovators. My personal favorite innovator is @ceeceeboom and her 10 biobanks 👀
$DAWN acquisition by Servier for $2.5bn is quite the ending for Ojemda. Who remembers this RAF inhibitor's bizarre & convoluted history? $BIIB $TAK https://t.co/uD5drO9ymr
The article outlines a four‑step framework for specialty pharma companies to redesign market‑access strategies as they move from approval to commercial launch. It stresses securing payer formulary placement, deploying a focused sales force, establishing a trusted pharmacy network, and centering...
Jake puts it crisply: >If discovery and early development move abroad, the nation risks locking in a massive trade imbalance: Foreign countries develop the drugs, and Americans pay the bills.
Eli Lilly has introduced Employer Connect, a new platform that partners with more than fifteen independent administrators to provide cost‑transparent access to its obesity drug Zepbound for U.S. employees. The service is designed to close the insurance coverage gap affecting roughly...
The latest generation of AI‑driven tools is transforming clinical trial data management by automating the build of electronic data capture (EDC) systems. Solutions such as CRScube’s AI‑led EDC builder can convert a PDF protocol into a structured database in hours...
Johnson & Johnson announced FDA approval of the Tecvayli (teclistamab) and Darzalex Faspro (subcutaneous daratumumab with hyaluronidase) combination for adults with relapsed/refractory multiple myeloma who have received at least one prior line of therapy. The approval is grounded in the Phase III...
Seqster has introduced 1‑Click DataLake, a real‑world data platform that aggregates anonymized electronic health‑record information from over 150 million patients and 200,000 clinicians across the United States. The solution delivers real‑time, longitudinal patient journeys to speed trial design, feasibility assessments, and...
Liberate Bio announced it has secured both exclusive and non‑exclusive licenses for patents covering chimeric antigen receptor (CAR) designs specifically engineered for myeloid cells such as monocytes and macrophages. The patents, obtained from Carisma Therapeutics and the University of Pennsylvania,...
The pharmaceutical industry has finally delivered an oral GLP‑1 weight‑loss pill, with Novo Nordisk launching an oral version of Wegovy earlier this year. Eli Lilly’s oral GLP‑1 candidate, orforglipron, is expected to receive approval imminently. Oral formulations overcome the injection barrier that...
Kainova Therapeutics, formerly Domain Therapeutics, announced a Series B funding round to advance its next‑generation GPCR‑modulating platform aimed at reversing tumor immunosuppression. The rebrand signals a strategic shift toward a broader oncology focus, leveraging GPCR pathways that control immune cell trafficking....
Harrison.ai has received U.S. FDA 510(k) clearance for its Acute Infarct Triage software, which analyzes non‑contrast CT brain scans to identify acute ischemic strokes. In validation studies the algorithm achieved roughly 89% sensitivity on thin‑slice CT and 86% on thicker...
Roche and Zealand Pharma disclosed Phase‑II (ZUPREME‑1) results for petrelintide, an amylin‑analog injected weekly, in 493 overweight or obese adults (mean BMI 37 kg/m²) with weight‑related comorbidities. The trial evaluated five dose levels against placebo over 42 weeks and met its primary endpoint,...
Ergostane steroid, as one of the major contributor to cranberry derived extracellular vesicle nanoparticles, restores ovarian function of murine premature ovarian failure https://t.co/CFcjDvzMnU https://t.co/le7VTHjEKa
Biopharma R&D is witnessing a rapid influx of specialized AI models for tasks such as structure prediction, retrosynthesis, and image analysis. While each tool delivers measurable benefits, their isolated deployment creates fragmented data streams, hidden costs, and increased cognitive load...
