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
The U.S. Food and Drug Administration issued a warning to ImmunityBio after a television ad and a podcast featuring billionaire biotech entrepreneur Patrick Soon‑Shiong suggested that the company’s bladder‑cancer drug Anktiva could cure or prevent all cancers. The FDA said the messaging created a misleading impression, violating federal advertising rules that restrict claims to approved indications. ImmunityBio has been asked to correct the material and submit a compliance plan. The agency’s action underscores heightened scrutiny of biotech promotional practices.
Optibrium has released a new PyMOL plugin that adds a graphical user interface to its QuanSA ligand‑based affinity prediction tool. QuanSA uses physically‑motivated machine learning to deliver free‑energy perturbation‑level accuracy without needing a protein structure, dramatically lowering computational cost. The...
Karyopharm reported mixed results from its Phase 3 SENTRY trial of selinexor in myelofibrosis, achieving a statistically significant reduction in spleen volume but raising safety concerns. The data fell short of expectations for overall survival benefit. Meanwhile, Rezolute announced it will...
T‑cell engagers are bispecific antibodies that redirect T cells to destroy cancer cells and are being explored for autoimmune disorders. Seven companies are leading the field: Adaptin Bio secured FDA IND clearance for APTN‑101 in glioblastoma; Candid Therapeutics merged with...
Amylyx Pharmaceuticals announced that the pivotal Phase 3 LUCIDITY trial of avexitide has completed enrollment, randomizing and dosing the final participant to bring the total to 78 patients across 21 U.S. sites. The double‑blind, placebo‑controlled study evaluates avexitide, a first‑in‑class GLP‑1...
Swiss drugmaker Novartis announced a $480 million investment to expand its manufacturing and R&D footprint in China. The plan allocates about $218 million to upgrade its Beijing Changping facility, which can produce up to 3 billion tablets annually, and $262 million to expand its...
Nutritionists and obesity physicians say GLP‑1 drugs such as Wegovy and Zepbound only deliver lasting weight loss when paired with a high‑protein, high‑fiber, well‑hydrated diet. With roughly one in eight U.S. adults on these medicines and more than 600,000 Wegovy‑pill...
A senior Trump administration health official warned that the president’s most‑favored‑nation (MFN) drug‑pricing strategy may unintentionally raise prices abroad rather than lower them in the United States. The flaw arises because manufacturers could postpone launching new drugs in foreign markets,...
This book explains how disruptive technologies and emerging trends such as robotics, artificial intelligence, 3D printing, precision medicine or patient design will impact the manufacturing and distribution of pharmaceuticals in order to prepare successfully for a better future of healthcare. Everything...
Regulatory agencies have launched inspections of 49 firms suspected of distributing unauthorized weight‑loss drugs. The sweep underscores heightened scrutiny of the booming weight‑loss market and raises questions about compliance across the industry.
Aardvark Therapeutics has paused two Phase 2 obesity trials, POWER and STRENGTH, after cardiac safety signals emerged in its lead compounds ARD‑101 and ARD‑201. The anomalies were observed in healthy volunteers receiving double the target dose, prompting a halt to the...
ImmuneBridge announced a $7.7 million second seed round, bringing its total seed capital to roughly $20 million, to commercialize its proprietary cell‑therapy screening and manufacturing platform for external partners. The platform combines machine‑learning‑driven donor selection with a unique small‑molecule that preserves stem‑cell...
Scientists at the U.S. Army Medical Research Institute of Infectious Diseases have engineered a dual‑action antibody that simultaneously targets two stages of Venezuelan equine encephalitis virus (VEEV) entry. The single‑molecule treatment protected animals even when given after exposure and neutralized...
Celosia Therapeutics has dosed the first participant in its Phase 1b KOANEWA trial of CTx1000, a gene‑editing medicine that clears pathological TDP‑43 protein in ALS patients. The open‑label study, run at Macquarie University Hospital, marks the first human test of...
Viz.ai and Alnylam Pharmaceuticals have teamed up to launch an AI‑driven care pathway targeting cardiac amyloidosis, a frequently underdiagnosed heart disease. The solution embeds the FDA‑cleared Us2.ai echocardiography algorithm and generative AI into hospital IT systems to automatically detect subtle...
FluoGuide A/S announced that the U.S. FDA has granted Fast Track designation to its intraoperative imaging agent FG001, targeting high‑grade glioma surgery. The designation recognizes the serious nature of glioblastoma and the agent's promise to improve tumor visualization during resection....
Atopic dermatitis landscape from Wedbush. Looks like there are two parallel vectors: lower injection frequency ( $APGE ) and combining new mechanisms with IL13/IL4R. So far $JNJ's IL13+IL31 failed (the only one to be directly compared to dupi) while $PFE's...
Quotient Therapeutics and Merck have signed a multi‑year collaboration to use Quotient’s somatic genomics platform for discovering new drug targets in inflammatory bowel disease. The agreement provides Quotient with $20 million upfront and includes milestone payments that could lift the total...
Artificial intelligence is reshaping the pharmaceutical and life‑sciences value chain, from molecule discovery with generative and structure‑prediction models to clinical trial design, manufacturing, regulatory submissions, intellectual property and supply‑chain orchestration. Tools such as AlphaFold and de‑novo design engines accelerate lead...
Radiopharmaceutical clinical trials are becoming a high‑velocity segment in 2026, but they remain vulnerable to three predictable bottlenecks: isotope supply chain fragility, imaging variability, and regulatory pathway selection. Axcellant, a boutique CRO with an integrated imaging core lab, demonstrates that...
Artis BioSolutions, a San Diego‑based advanced therapies firm, has launched a synthetic DNA manufacturing hub in Boston using Syngoi Technologies' proprietary enzymatic platform. The new site complements its GMP manufacturing facility in Watertown, creating a bi‑continental network with an existing...
A bipartisan congressional commission focused on maintaining U.S. leadership in biotechnology will host a meeting with leading biotech investors next week. The investors, representing venture capital firms and industry funds, aim to discuss policy gaps, funding mechanisms, and regulatory hurdles....
Prestige Biopharma announced topline results from its phase‑III SAMSON‑II trial comparing HD204, a biosimilar to Avastin (bevacizumab), with the reference product in 625 patients with advanced non‑squamous non‑small cell lung cancer. The study demonstrated an objective response rate (ORR) of...
Mosaic Therapeutics will present preclinical data on its lead program MOS101—a combination of the next‑generation MDM2 antagonist ASTX295 and the FDA‑approved PARP inhibitor olaparib—at the AACR 2026 Annual Meeting. The poster focuses on BRCA2‑mutant, TP53‑wild‑type solid tumours and highlights the...
Japanese pharmaceutical company Shionogi is buying out Apnimed’s 50% stake in their sleep‑disorder joint venture for an upfront payment of $100 million. The acquisition gives Shionogi full control over the partnership’s pipeline, which focuses on treatments for conditions such as obstructive...
The U.S. Food and Drug Administration approved a 7.2‑milligram version of Novo Nordisk’s semaglutide weight‑loss drug Wegovy, dubbed Wegovy HD. Clinical data showed participants lost roughly 19% of body weight, about 47 pounds on average, compared with 16% on the...
Gubra and AbbVie announced that participants in a Phase 1 multiple‑ascending‑dose study of ABBV-295 lost between 7.8% and 9.8% of their body weight after roughly three months. The amylin analog was well tolerated, positioning it as a potential new class of...
India's high court cleared Dr Reddys to produce the weight‑loss drug semaglutide, unlocking a billion‑dollar market, as health regulators in Ireland recalled 18,000 Philips Respironics machines over foam degradation. The twin moves underscore mounting compliance pressure across the pharma and...
The FDA has broadened approval for Imcivree (setmelanotide), creating the first targeted therapy for acquired hypothalamic obesity and reporting an 18.4% placebo‑adjusted BMI reduction in Phase III trials. Parallel research shows that deploying pharmacy technicians as vaccine injectors dramatically improves uptake...
CMS will require pharmaceutical manufacturers to submit the "reasonable assumptions" used to calculate their quarterly Average Sales Price (ASP) beginning in 2026. The move formalizes the estimation process that underlies ASP, covering rebates, bundled discounts, free‑goods programs, 340B sales and...
Please. This is nonsense. You figured out a way to swap castor oil for albumin making it easier to administer paclitaxel, a standard chemotherapy. Same side effects. Look at the label. The best thing you did, for you, was secure...
Researchers discovered that clemastine fumarate activates lipophagy in oligodendrocyte progenitor cells (OPCs), clearing lipid droplets that impede differentiation. In vitro, the drug enhanced OPC maturation and removed myelin debris, while in a cuprizone‑induced mouse model it restored myelin integrity and...
We are very happy to join forces together with the wonderful ASKA to go after Novel Targets for Women's Health. Gynecological diseases have long posed challenges, including difficult diagnosis, limited treatment options, and a substantial disease burden, affecting millions of...
A recent British Journal of Ophthalmology study found that users of Wegovy, the higher‑dose GLP‑1 weight‑loss injection, face a five‑fold increased risk of non‑arteritic anterior ischemic optic neuropathy (NAION), an eye‑stroke that can cause blindness. The condition occurs in roughly...
China's National Medical Products Administration approved Sciwind Biosciences' Ecnoglutide injection, marking the country's fifth GLP‑1 obesity therapy. The drug demonstrated a 15.4% average weight loss in the Phase III SLIMMER trial, with over 90% of participants achieving at least 5%...
The 9th Annual Corporate Compliance & Transparency in Life Sciences Conference convenes more than 20 compliance leaders, legal experts, and industry innovators for a single-stage event. Attendees will experience over 15 hands‑on presentations and case studies, targeting actionable strategies for...
Blood levels of the Alzheimer's biomarker p-tau217 may help identify which women are more vulnerable to dementia when using combined hormone therapy after menopause, while estrogen-only therapy does not show the same association. menopause
The free market (no government-granted patent monopolies) will make Ozempic affordable for tens of millions of people https://t.co/SYl5M82mux
Accumulus Technologies launched the Accumulus Connector, extending its platform into customers’ existing pharma and biotech systems for live, bi‑directional regulatory communication. The Connector provides real‑time, one‑click global submissions to more than 70 national regulators, eliminating manual data reconciliation. By integrating...
SWIFT-seq enables comprehensive single-cell transcriptomic profiling of CTCs in multiple myeloma & precursors [Aug 8, 2025] Lightbody, @RomanosSP et al. @IrenemGhobrial - @LabGhobrial @NatureCancer https://t.co/LhqmRShFo9 #mmsm #LiquidBiopsy #cactc THREAD: https://t.co/87VZtDNy80 https://t.co/41MRROCXwI
#mmMRD healthcare social media hashtag [Nov 21, 2020] Minimal/measurable residual disease (MRD) in multiple myeloma. Submitted by @bdermanmd to @symplur @healthhashtags https://t.co/grkkiox2yh #mmsm No longer available at Symplur https://t.co/WmKH0fcB4p
Evotec SE announced that its Seattle CDMO, Just‑Evotec Biologics, has been selected by BARDA’s BioMaP‑Consortium for a multi‑year, up‑to‑$10 million program to optimize manufacturing of a monoclonal‑antibody cocktail against Ebola and Sudan viruses. The same day, Evotec disclosed that partner Bristol...
How former Acorda CEO Ron Cohen landed at a Parkinson’s cell therapy startup https://t.co/93JYvJGM9I by @realJacobBell #biotech #startups
President Donald Trump and Japanese Prime Minister Sanae Takaichi discussed a $40 billion U.S. nuclear investment and pledged cooperation on drug‑pricing reform. Japan plans a 4% cut to total drug spending, further lowering reimbursements for new medicines. The article warns that...
A potentially game-changing discovery by @UCSF's Justin Eyquem @j_eyquem & colleagues – injecting cancer-fighting immune cells directly into the body kills several types of cancers in mice. Paper in @Nature: https://t.co/EXbsfx7whl Summary/video by UCSF: https://t.co/qLYXXvEDTV
Enlicitide, a once‑daily oral PCSK9 inhibitor, lowered LDL cholesterol by as much as 60% in a phase 3 trial of 2,909 high‑risk adults. The results, published in The New England Journal of Medicine, could shift cholesterol management from injections to...
A market analyst recommends Pfizer (PFE) and UnitedHealth Group (UNH) as long‑term holdings despite recent market swings. The stocks trade at roughly 9x and 15x forward earnings, respectively, offering perceived discounts in a climate of gold price crashes, rising energy...
Bausch + Lomb announced that its implant‑free ELIOS glaucoma treatment system met both primary efficacy endpoints in a 24‑month pivotal study, with 76% of patients achieving a 20% intraocular pressure reduction and 82% remaining medication‑free. The news sent the company’s shares up...
Bay Area biotech R3 Bio announced a plan to grow nonsentient “organ sacks” as a replacement for monkey testing, with backing from Singapore’s Immortal Dragons fund. The approach could reshape pre‑clinical drug safety studies if it proves scalable.
A recent Molecular Psychiatry review highlights anesthetics as a promising new class of therapeutics for post‑traumatic stress disorder (PTSD). It outlines how NMDA‑receptor antagonists, α2‑adrenergic agonists, GABA‑A modulators and certain opioids can modulate fear circuitry and memory reconsolidation. Pre‑clinical models...
