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
Philadelphia’s biotech ecosystem, long known for CAR‑T breakthroughs, is diversifying into obesity, metabolic disease, gene editing, and solid‑tumor immunotherapies. Nine local companies illustrate this shift, from Alveus Therapeutics’ $197 million Series A obesity program to Cabaletta Bio’s autologous CD19 CAR‑T for autoimmune disorders. Others such as Century Therapeutics and Dispatch Bio have secured IND clearances and sizable financing to advance iPSC‑derived beta‑cells and a viral‑vector‑primed CAR‑T combo, respectively. The region’s academic roots continue to fuel rapid translational progress and attract substantial capital.
February 2026 saw a surge of biosimilar activity, with key regulatory approvals for products such as Sandoz Enzeevu (Eylea), Accord Filkri (Neupogen), and STADA Gotenfia (Simponi) across the US, Canada and the EU. Companies forged new licensing and commercialization deals that extend biosimilar...
Quell Therapeutics has launched the phase 1/2 CHILL trial of its autologous CAR‑Treg therapy QEL‑005 in rheumatoid arthritis and systemic sclerosis, after pausing the liver‑transplant program QEL‑001. The multi‑center study will enroll patients in the UK, Germany and Spain, with read‑out...
Boehringer Ingelheim has terminated its metabolic dysfunction‑associated steatohepatitis (MASH) collaboration with OSE Immunotherapeutics after an exploratory Phase 2 trial failed to meet its primary endpoint. The original 2018 deal included a €15 million upfront payment and up to €1.1 billion in milestone potential,...
In this episode, Bruno Quinney highlights four major developments: a long‑acting injectable HIV regimen (cabotegravir + rilpivirine) that cut virological failure risk by nearly half versus daily oral therapy; SolasCure’s ORES wound gel, which accelerated debridement 22‑fold and healing seven‑fold in chronic...
Lynk Pharmaceuticals announced topline Phase III data for its oral JAK inhibitor zemprocitinib in 356 patients with moderate‑to‑severe atopic dermatitis. Both the 12 mg and 24 mg doses met the co‑primary endpoints at week 16, delivering 38‑46 percentage‑point improvements in EASI‑75 and roughly 30 percentage‑point gains...
The GAO report reveals that, despite a conditional approval pathway introduced in 2004 and expanded in 2018, FDA‑approved animal drugs for minor species and niche uses remain scarce. From FY2018 through FY2025, only 13 drugs received conditional approval, all targeting...
New reporting from me taking you inside the $QURE - FDA Huntington's gene therapy imbroglio. I spoke to a senior FDA official late yesterday, who told me agency reviewers "are not convinced there’s any therapeutic benefit of the product. If...
The FDA concluded that UniQure’s experimental gene therapy for Huntington’s disease has not demonstrated therapeutic benefit based on existing clinical data. Reviewers said they are not persuaded by the evidence, prompting the agency to block the company from submitting a...
Alzheimer’s research is increasingly looking beyond plaques and tangles to the brain’s immune response. A study published in Elsevier's Pharmacological Research journal used AI to screen 1.6M compounds for a Galectin-3 (Gal-3) blocker that could cross the brain barrier. In Alzheimer’s-like...
T-cell engagers for autoimmunity. Listen to Ken Song describe the opportunity as CEO of San Diego-based Candid Therapeutics. Sponsored by @AlphaSenseInc & Dash Bio. https://t.co/Ct2viH00c4
Innovent’s pirtobrutinib, marketed as Jaypirca, received Chinese NMPA approval for adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma who have undergone at least one prior therapy, including a BTK inhibitor. The decision follows the phase‑III BRUIN CLL‑321 trial,...
$APGE should can the OX40 bispec IMO. You’re basically just tacking on a black box with that MOA at this point…
The cGAS‑STING pathway, a DNA‑sensing immune circuit, becomes aberrantly activated in the aging brain as mitochondrial and nuclear DNA escape into the cytoplasm. This chronic activation drives low‑grade neuroinflammation, contributing to neurodegenerative diseases such as Alzheimer’s, Parkinson’s and ALS. Preclinical...
Patent term extension (PTE) offers up to five extra years of exclusivity for FDA‑approved drugs, but biologics present a unique hurdle because the law hinges on defining the “active ingredient.” Unlike small molecules, biologics are large, variable structures, making it...
The U.S. Centers for Medicare & Medicaid Services (CMS) has pushed back the deadline for manufacturers to join its GENEROUS pilot, extending the initial cut‑off to April 30 while keeping the final deadline at June 30. The voluntary program applies a Most‑Favoured...
Eight leading European microphysiological systems companies have launched the Industry Alliance for Microphysiological Systems (IAMPS), the world’s first trade association dedicated to MPS technologies. IAMPS will represent organ‑on‑chip, organoid and related NAM developers, aiming to harmonize standards, promote data sharing,...
Merck and Eisai disclosed Phase III LITESPARK-011 results showing that the oral combination of Welireg (belzutifan) and Lenvima (lenvatinib) outperformed cabozantinib in 747 patients with advanced renal cell carcinoma who progressed after anti‑PD‑1/PD‑L1 therapy. At a median follow‑up of 29...
Dermatology research is booming, with more than 16,000 trials completed, ongoing, or planned by the end of 2025. The Asia‑Pacific region now hosts roughly 8,000 studies, a five‑fold increase over the past decade, matching North America and Europe combined. Low...
Researchers at Vilnius University used cryogenic electron microscopy to map eleven CRISPR‑Cas protein complexes, including three variants of a newly described Cas9‑Cas1‑Cas2‑Csn2 supercomplex. The study shows that Cas9, traditionally viewed only as a DNA‑cutting enzyme, also directs the selection and...
Researchers demonstrated that a clinically‑available 1.5 T static magnetic field can trigger the release of encapsulated molecules from magnetoliposomes (MLs). The study used citric‑acid‑stabilized Fe₃O₄ nanoparticles, with and without a chitosan coating, and monitored structural changes via SAXS and DLS. Fluorescence...
Earlier this year the UK government pledged £82.6 million to AI‑focused drug‑discovery firms, underscoring its ambition to lead the global digital health transformation. Major players such as AstraZeneca are launching dedicated health‑tech units like Evinova to accelerate clinical‑trial design, while the...
North Carolina’s Holly Springs is becoming a hub for next‑generation obesity drugs as Amgen and Roche commit more than $3.5 billion to build manufacturing capacity. Amgen’s $1.55 billion rollout includes a new drug‑substance plant and a second facility slated for 2032, while Roche’s...
Omega‑3 supplementation trials produce mixed results due to differences in dose, population risk, and chosen endpoints. High‑dose EPA/DHA studies in secondary‑prevention cohorts with elevated triglycerides, such as REDUCE‑IT, show significant cardiovascular benefit, whereas lower‑dose primary‑prevention trials like VITAL and ASCEND...
After a wave of senior departures and a 3,500‑position cut, the FDA entered 2026 with a largely inexperienced workforce, raising uncertainty for drug developers. The agency’s new AI guidance, released in January, outlines best practices but emphasizes human oversight and...
CVRx reported fourth‑quarter 2025 revenue of $16 million, a modest 4% year‑over‑year increase, while gross margin improved to 86% driven by higher selling prices and manufacturing efficiencies. Net loss widened to $11.9 million as SG&A and R&D expenses outpaced revenue growth, but...
Cumberland Pharmaceuticals reported fiscal 2025 revenue of $44.5 million, an 18% increase year‑over‑year, driven by a 31% Q4 surge to $13.7 million from Vibativ, Sancuso and the newly launched Talicia. Adjusted earnings turned positive at $1.7 million and operating cash flow rose to...
The FDA’s Center for Drug Evaluation and Research (CDER) has published its annual New Drug Application (NDA) and Biologics License Application (BLA) approval reports for calendar years 2015 through 2025, each linked in a consolidated list. These reports detail the...
In a quick‑bite interview at the February 2026 VIVE conference, Miriam Paramore discussed RxUtility, a health‑tech platform that consolidates manufacturer coupons and cash‑price data to present consumers with the lowest possible drug price at the point of dispensing. The solution...
A head‑to‑head study of 55 post‑prostatectomy patients compared two FDA‑cleared PSMA PET agents, Posluma (flotufolastat F‑18) and Pylarify (piflufolastat F‑18). Posluma demonstrated markedly lower bladder radioactivity, with a median bladder SUV of 10.9 versus 29 for Pylarify. This reduction translated into higher...
Scientists have identified a molecular checkpoint that guides planarian stem cells during regeneration. The roundabout A receptor (RoboA) suppresses inappropriate pharyngeal differentiation by regulating the transcription factor FoxA, while the extracellular protein Anosmin1a partners with RoboA to fine‑tune cell identity...
The FDA has mandated electronic submissions to the FAERS database using the ICH‑endorsed E2B(R3) standard, beginning January 16 2024 for post‑marketing drug and biologic reports and April 1 2024 for pre‑marketing IND safety reports. Companies have until April 1 2026 to transition fully, after which only...
Vanda Pharmaceuticals received FDA approval for Bysanti (milsaperidone) to treat acute manic or mixed episodes of bipolar I disorder and schizophrenia in adults. The approval introduces a new chemical entity in the atypical antipsychotic class that is bioequivalent to Vanda’s...
The FDA Adverse Event Reporting System (FAERS) is a public database that collects adverse event, medication error, and product quality complaint reports for drugs and biologics after market approval. It follows the ICH E2B international safety reporting guidance and uses...
Johnson & Johnson announced a more than $1 billion investment to build a next‑generation cell and gene therapy manufacturing complex on a 154‑acre site in Montgomery County, Pennsylvania. Construction is slated to start in the second half of 2026, creating over...
The breakthrough blood test p-tau 217 for risk and diagnosis of Alzheimer's disease is more accurate than total p-tau 217, in a head-to-head comparison using PET imaging https://t.co/faILpVr9Ua https://t.co/lzUgFnFUP5
Boehringer Ingelheim’s oral kinase inhibitor Hernexeos received FDA accelerated approval for first‑line HER2‑mutant lung cancer in a record‑fast 44 days, thanks to the National Priority Voucher program. The drug demonstrated a 76 % response rate, positioning it as a breakthrough in personalized...
Idiopathic pulmonary fibrosis (IPF) continues to pose a severe, often late‑diagnosed respiratory challenge, with mortality exceeding many cancers. Boehringer Ingelheim’s Martin Beck highlighted the shift toward earlier detection using AI‑enhanced imaging and a broader view of IPF as a heterogeneous,...
Arizona State University researchers quantified how water interacts with biomolecule‑coated magnetite nanoparticles, revealing that surface coatings dramatically reshape hydration energetics, immune recognition, and drug‑delivery performance. Using a calorimetry‑gas adsorption system, they measured water adsorption on particles coated with bovine serum...
Generate Biomedicines priced its IPO at $16 per share, raising $400 million to fund pivotal trials of its lead candidate GB‑0895. The antibody blocks the TSLP pathway and is engineered for six‑month dosing, a potential advantage over existing asthma biologics that...
The FDA issued a final guidance document outlining best practices for conducting and reporting pharmacoepidemiologic safety studies that rely on electronic healthcare data, such as administrative claims and electronic medical records. It prescribes how sponsors should document study design, analytical...
Seattle Stem Cell Center was ordered to pay $24 million after a jury found the clinic liable for the 2019 death of Michael Trujillo, who suffered catastrophic bleeding following an undocumented epidural injection while on blood‑thinning medication. Evidence showed the procedure...
A systematic RCT analysis of GLP-1 drugs show they are more effective in women than men for weight loss https://t.co/cycwi4nnN5 https://t.co/KOl0FgKaZD
Roche pill succeeds in another MS study, but approval questions linger https://t.co/JY8uZCI5iA @ByJonGardner $RHHBY $SNY
Alnylam Canada received a positive recommendation from Canada’s Drug Agency to list AMVUTTRA® (vutrisiran) for public reimbursement in adult patients with cardiomyopathy caused by wild‑type or hereditary ATTR amyloidosis. The therapy, approved by Health Canada in December 2025, expands the...
UniQure says FDA wants another study of Huntington’s gene therapy https://t.co/1pgFDoE2V9 by @realJacobBell $QURE - 35% #GeneTherapy #Huntingtonsdisease
The European Medicines Agency’s CHMP has issued a positive opinion on Moderna’s mCombriax, a combined COVID‑19 and influenza mRNA vaccine, after a Phase III trial showed stronger immune responses than a mixed regimen of Sanofi’s flu shot and Spikevax. EMA approval...
At CROI 2026, researchers spotlighted the growing neurodegenerative burden among aging people living with HIV, emphasizing heightened risks of depression and cognitive vulnerability despite long‑term antiretroviral therapy. Parallelly, the University of Southern California announced a novel series of MAPT aggregation...
BioWorld’s “In the Clinic for March 2, 2026” page functions as a centralized gateway to the latest biopharma, med‑tech, and scientific content. It aggregates data snapshots, special reports, infographics, and market scorecards covering everything from GLP‑1 trends in China to mRNA vaccine...
On March 2, 2026 BioWorld published a regulatory snapshot covering biopharma and med‑tech firms such as AS Software, Asieris, Boehringer Ingelheim, Deephealth, Eli Lilly, Ipsen, Moderna, Neurogene, Novartis, Optellum, Photocure, Regeneron, Sanofi, Sentynl, Synergy Spine Solutions and X4. The roundup highlights...
