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
A recent double‑blind trial found that a combination of two GLP‑1 receptor agonists cut fat mass significantly while sparing lean muscle in adults with obesity. Over 24 weeks, participants lost an average of 5 % body fat, with lean mass decline limited to less than 1 %. The study involved 300 participants and reported safety comparable to monotherapy, with mainly mild gastrointestinal side effects. These findings point to a synergistic benefit of dual GLP‑1 therapy for weight management.
Boosted Brain Cells Erase Dementia-Linked Proteins “At nearly six months of age, when untreated mice normally have brains saturated with harmful plaques, brains of treated mice were plaque-free. Meanwhile, older mice with plaque-saturated brains at the time of treatment saw a...
Helus Pharma reported Phase II data for its psychedelic candidate HLP004, indicating symptom improvement in generalized anxiety disorder patients for up to six months. However, the trial’s low‑dose active control arm showed similar efficacy, making it difficult to isolate the...
Helus Pharma reported Phase II data for its psychedelic candidate HLP004 in generalized anxiety disorder, showing symptom improvement lasting six months. However, the trial’s active low‑dose control produced outcomes similar to the therapeutic dose, making it difficult to isolate the drug’s...
Researchers from Kyoto University and INSERM identified tanycytes as a previously unknown conduit that clears tau protein from cerebrospinal fluid into the bloodstream. In rodent and cellular models, blocking vesicular transport in these cells dramatically slowed tau efflux and worsened...
Australian biotech firm CSL Seqirus has secured a contract with Canada’s Public Health Agency to deliver up to 15 million doses of a cell‑based, adjuvanted pandemic influenza vaccine if the WHO declares a pandemic. The doses will be manufactured at CSL...
UT San Antonio has been given a $38biin ARPA-H grant to study the health benefit effects of three drugs in humans, namely, rapamycin, dapagliflozin and semaglutide …🧵 https://t.co/fAX14ngoyD
![[Comment] Pimicotinib: A New Agent for an Orphan Disease](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/5b160e8a2b2821c82aa66047ca629679.webp)
Tenosynovial giant cell tumour (TGCT) is a rare, locally aggressive neoplasm driven by CSF‑1 over‑expression. The phase 3 MANEUVER trial evaluated pimicotinib, a selective CSF1R kinase inhibitor, against placebo in patients with inoperable or relapsed diffuse‑type TGCT. Results demonstrated statistically significant...
![[Correspondence] Changes in Paracetamol and Leucovorin Use After a White House Briefing](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/59b0c8a4048a3d105c9221755df21495.webp)
The White House briefing on Sept 22 2025 linking prenatal acetaminophen to autism and promoting leucovorin was followed by an interrupted‑time‑series analysis of the Epic Cosmos database. Emergency‑department orders for paracetamol among pregnant women fell 10% (OER 0.90) while use among non‑pregnant women showed...
Asian biotech firms outside China announced 72 deals in 2024‑25, emphasizing first‑in‑class assets over me‑better or me‑too programs. At least four transactions involve novel targets such as CNTN4, GPR52, MTARC1 and OK‑1, spanning new modalities and indications. Global pharmaceutical companies...
A new observational study of nearly 22,000 chronic‑migraine patients found that those who started GLP‑1 receptor agonists for diabetes or obesity experienced fewer severe migraine events than peers on the preventive drug topiramide. Over a 12‑month follow‑up, GLP‑1 users had...
Life‑science marketers face mounting pressure to deliver personalized content while meeting stricter privacy regulations such as CCPA and the EU Data Act. Companies are shifting from static consent checkboxes to unified, real‑time consent frameworks that synchronize preferences across CRM, CDP,...
Biotech firms are cutting staff as venture‑capital inflows dry up, clinical‑trial setbacks mount, and operating costs climb. While breakthroughs in gene therapy, RNA therapeutics and precision medicine continue, companies are trimming non‑core programs to preserve cash. Strategic restructurings aim to...
The FDA issued a Request for Information (RFI) on March 5, 2026, asking for public input about new standards for in‑home opioid disposal products. The agency is evaluating whether opioid manufacturers should be required to supply disposal systems directly through dispensers....
Scientists delivered naked Cldn11 messenger RNA directly into the testes of genetically infertile male mice, restoring Sertoli cell function and enabling spermatogenesis. The treatment produced viable sperm that generated healthy offspring via in‑vitro fertilization, without permanent germline alteration. The approach...
The FDA’s FY 2025 Generic Drug Program report details progress on abbreviated new drug applications (ANDAs) under priority review and competitive generic therapy (CGT) designations. By the fourth quarter, pending priority‑review ANDAs fell to 40, while CGT ANDAs awaiting FDA action...
Schrödinger’s stock has plunged 88% over five years, leaving a sub‑$1 billion market cap. The company’s AI‑driven software segment posted 11% revenue growth in 2025 and aims for modest 12% growth in 2026, while shifting customers to cloud contracts that compress...
The FDA released a comprehensive Patient‑Focused Drug Development (PFDD) glossary to standardize terminology across its guidance documents mandated by the 21st Century Cures Act and PDUFA VI. The glossary defines key concepts such as attributes, benefit‑risk assessment, clinical outcome assessments, patient‑reported...
Gül Dölen, a leading neuroscientist at UC Berkeley and Johns Hopkins, discussed the transformative potential of psychedelic‑assisted therapy during the 2025 Aspen Ideas Festival. Her research demonstrates that compounds such as psilocybin and MDMA can rapidly alleviate treatment‑resistant depression, complex PTSD,...
Cognito Therapeutics closed an oversubscribed $105 million Series C round to fast‑track its neurodegenerative programs, while Nexcure raised $19 million in a Series A to fund its immunology pipeline. At the same time, artificial‑intelligence advances—particularly large language models—are reshaping pharmacovigilance by automating analysis of...
British biotech Vesalic reports a breakthrough that links amyotrophic lateral sclerosis (ALS) to a systemic metabolic dysfunction detectable in blood extracellular vesicles (EVs). The company says the EVs carry a toxic lipid cargo that harms motor neurons, and its blood‑based...
At the recent ASCO‑GU meeting, industry leaders highlighted that the greatest threat to emerging bladder‑cancer programs is strategic, not clinical. Phase‑2 candidates are poised to enter Phase‑3 trials, but shifting control arms, evolving endpoints, and changing patient demographics risk rendering...
Psychedelic antidepressants are poised for FDA review this year, driven by strong investor and patient interest. William Blair analysts note that while Johnson & Johnson’s Spravato generated $1.7 billion in 2025 sales, psychedelics are unlikely to capture the entire treatment‑resistant depression market. Companies...
Alnylam Pharmaceuticals has signed a deal with Tenaya Therapeutics, providing $10 million upfront and the potential for up to $1.13 billion in milestones to discover up to 15 new genetic targets for heart disease. Tenaya will apply its modality‑agnostic platform to validate...
UniQure’s one‑time gene therapy AMT‑130 showed a 75% slowdown in Huntington’s disease progression in its Phase 1/2 trial, prompting expectations for a BLA filing in early 2026. The FDA, however, reversed its earlier stance and now requires a sham‑controlled Phase 3 study,...
Amazing quote in this NYT story on the FDA from @By_CJewett. Janet Woodcock, a top drug regulator for decades, said reversals for a promising therapy send shock waves through the industry. “The Huntington’s refusal I thought was truly evil,” she told NYT....
Thermo Fisher Scientific has opened a Cryo‑Electron Microscopy Drug Discovery Center in South San Francisco, offering pharmaceutical and biotech firms direct, hands‑on access to cutting‑edge cryo‑EM instrumentation. The facility is designed to accelerate structural insight generation, enabling faster, more cost‑effective...
The CNBC Cures Summit opened with Becky Quick urging families and innovators to accelerate rare‑disease research. Speakers highlighted a widening gap between rapid scientific breakthroughs—gene therapies, AI‑driven diagnostics, and modular “nodal biology”—and an aging regulatory framework. Leaders from Biogen, the...
My friend @adamfeuerstein delivers some great reporting and smart commentary on the FDA's direction that $QURE conduct a new randomized trial for its Huntington's drug. A senior FDA official and the company disagree on the basic question of whether the...
PepGen’s Phase 2 FREEDOM2 trial in myotonic dystrophy type 1 received a partial FDA clinical hold due to concerns over a sub‑chronic mouse study that showed blood‑pressure changes. The agency did not question the Phase 1 human data, and the company continues dosing...
Randomized trials for non‑small cell lung cancer often miss real‑world nuances that affect patients' quality of life. Real‑world data (RWD) and social determinants of health (SDOH) expose hidden barriers such as transportation gaps and limited molecular testing access. Pharma can...
Roche announced a $480 million, five‑year investment in South Korea to build a national clinical‑trial ecosystem, fund R&D infrastructure, and train specialized personnel. The pledge also includes support for domestic biotech startups aiming for global markets. This move aligns with a...
A new BMJ meta‑analysis of over 600,000 U.S. veterans with type‑2 diabetes found that patients prescribed GLP‑1 agonists such as semaglutide or tirzepatide were 14% less likely to develop substance‑use disorders (SUD) than those on SGLT2 inhibitors. Over a three‑year...
Late 2025 saw the FDA grant traditional approval to pirtobrutinib, an oral, reversible BTK inhibitor targeting multiple B‑cell malignancies. The drug demonstrated robust efficacy in BTK‑resistant chronic lymphocytic leukemia and small lymphocytic lymphoma, backed by positive Phase 3 data and early...
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE...
Ribosomes as Drug Targets. The Quiet Rewrite of Psychiatry and Beyond. | Ep. 950 https://t.co/gKlWpzO1Ci https://t.co/ImGOmUVrSO
Airiver Medical announced that the U.S. Food and Drug Administration granted Breakthrough Device Designation to its pulmonary drug‑coated balloon (DCB) for treating central airway stenosis. The company also enrolled and treated the first patient in a pivotal trial that will...
stunning week in cell and gene therapy: in vivo base editing of PCSK9 delivered via LNP, w clinical data (Nat Med), preclinical POC of CAR-astrocytes engineered w AAV (Science) and of self-amplifying RNA delivered IM (also Science), Japan approves gene...
Doesn’t look like $LLY and $NVO have much to worry about from $RHHBY obesity 🤷🏻♂️
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to consume solid tumors from the inside out. The spores infiltrate the oxygen‑free tumor core, proliferate and break down cancerous tissue. By inserting an oxygen‑resistant gene and a quorum‑sensing...
Will we give a shot of protective, self-amplifying RNA in patients with a heart attack in the future? @ScienceMagazine an intriguing innovation https://t.co/FcJme1ElyU https://t.co/lABFRkR3qg
When cancer niches are rapidly changing they create both a vacuum of indecision and fresh opportunities for smart players. Here we explore bladder cancer and look at where the field is headed... https://t.co/cf93Qb8dhe https://t.co/fx5V1MVwYN
The UK government announced a new package of measures to accelerate space‑based pharmaceutical manufacturing, offering regulatory clarity and a sandbox for companies developing drugs in microgravity. The initiative, led by the Department for Science, Innovation and Technology, brings together the...
Lilly targets employers in new bid to broaden access to obesity drugs https://t.co/Y6fmcmTcf1 @ByJonGardner $LLY $NVO #obesity
China competition is coming for the US's multimillion-dollar gene therapies -- a must-read from @Jared_Whitlock about what's happening there and how it will affect access around the world: https://t.co/F7KaMv3Kkp
Targeted therapies are reshaping oncology, yet chemotherapy remains essential. Immune checkpoint inhibitors like Keytruda generate $31.7 bn sales in 2025 and cover more than 40 indications. The FDA granted 63 antibody‑drug conjugate review designations in 2024, nearly double the previous peak....
20 years of Yamanaka stem cell factor research culminates in 2 conditional approvals for heart disease and Parkinson's disease in Japan https://t.co/8oxKxXYafu by Shinya Yamanaka @CellStemCell https://t.co/U878IxSfSi @ScienceMagazine https://t.co/HCCPaTEP7e
I speculated last month that HMPL-A580 was an anti-EGFR ADC https://t.co/XFDtZfWWkH and $HCM just confirmed that this is the case. Relevant for $ALXO
5March: Back in 2021, I wrote about a significant round of layoffs at Theravance Biopharma ( $TBPH). The company is cutting jobs again, and has begun a strategic review, after a Phase III failure. Details in my latest for @GENbio:...
Erythrocyte–anti-PD1 conjugates in persons with advanced solid tumors resistant to anti-PD1/PDL1: preclinical characterization and results of a phase 1 trial https://t.co/1GQMlUHn4Y https://t.co/0it5nTvUm0
