Know What's Happening in Pharma
Know What's Happening in Pharma
FDA greenlights KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA approved KRESLADI, a gene‑editing treatment from Rocket Pharmaceuticals for severe leukocyte adhesion deficiency‑I in children lacking a bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine, highlighting CIRM’s role in translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
Lupin is pivoting toward proprietary new chemical entities (NCEs) in India, aiming to launch multiple innovative products by next year. The company plans to focus on cardiac, diabetes, respiratory and oncology molecules, leveraging lower development costs and faster time‑to‑market. AI is being integrated into its R&D to accelerate discovery, and Lupin will introduce a generic version of semaglutide as soon as patent protection expires. The firm reported revenues of ₹22,707 crore, underscoring the financial backing for this strategic shift.
The Enemy isn't Big Pharma. It's the FDA and Regulators who won't allow you to test experimental therapies on yourself (or your dog). Even if it's life or death.
The man engineering smarter, healthier babies before they're born: Jonathan Anomaly. This philosopher-turned-biotech founder says the future of your child's health starts at the embryo. Here are 9 things you need to know about embryo selection: https://t.co/XVQF5le4bE
Eli Lilly has launched Employer Connect, a platform that lets large employers purchase its weight‑loss injection Zepbound at a discounted $449 price, aiming to close the coverage gap that leaves over half of workers without obesity drug benefits. The program bypasses...
Japan has become the first country to grant conditional approval for two regenerative medicines that use induced pluripotent stem cells—AMCHEPRY for Parkinson’s disease and RiHEART for severe heart failure. The Parkinson’s therapy implants dopamine‑producing neurons into the brain, while the...
Researchers analyzing over 30 million adverse‑event reports found that patients using Wegovy, the high‑dose semaglutide injection for obesity, have about five times the odds of developing ischemic optic neuropathy (ION) compared with those on Ozempic, the lower‑dose diabetes formulation. The association...
Paul's story developing a cancer vaccine for Rosie inspired me. I'm going to build something so that EVERYONE can do the same thing. My dog Lady died of hemagiosarcoma and I wish I'd been able to save her too ETA - hopefully a...
A systematic review and network meta‑analysis of 32 randomized trials examined twelve hypnotic agents in adults with obstructive sleep apnea (OSA). The analysis found that most sleep‑inducing drugs do not worsen the apnea‑hypopnea index or oxygen saturation, challenging the long‑standing...
A phase‑3 BMJ trial reported that adding camrelizumab to standard chemoradiotherapy improved 36‑month progression‑free, distant‑metastasis‑free and locoregional‑recurrence‑free survival in high‑risk nasopharyngeal carcinoma. The protocol combined two concurrent cycles with 17 adjuvant cycles, yet only 61.9% of patients completed the full...
Zydus Lifesciences has received NMPA approval for its oral anaemia drug Desidustat, targeting renal anaemia in adult chronic kidney disease patients who are not on dialysis. The therapy, a hypoxia‑inducible factor‑prolyl hydroxylase inhibitor, demonstrated efficacy and safety in Chinese Phase III...
The first human age-reversal trial is officially happening. But before the FDA cleared it, Harvard professor David Sinclair had to pull off a mice experiment most scientists thought was impossible: "These mice had their optic nerve regenerated. We were able to...
AI: Short-Term Cliffs (0-3 years remaining): Drugs like sacubitril/valsartan, dapagliflozin, pembrolizumab, ocrelizumab, daratumumab, and apixaban face imminent generic/biosimilar pressure Mid-Term Protection (4-7 years): Risankizumab and dupilumab have solid runway, supported by ongoing label expansions. Long-Term (8+ years): Tirzepatide's robust patents position it...
YolTech Therapeutics announced FDA approval of its IND for YOLT-202, an in vivo adenine base‑editing therapy targeting Alpha‑1 Antitrypsin Deficiency. The clearance permits an open‑label, single‑dose Phase 2/3 expansion study across the U.S. and other regions. In the ongoing first‑in‑human trial,...
Cambridge chemists have unveiled a light‑driven “anti‑Friedel‑Crafts” reaction that forms carbon‑carbon bonds using only LED illumination at ambient temperature. The metal‑free method allows precise, late‑stage modifications of complex drug molecules, cutting months of multistep synthesis. Tested on a broad set...
Pretzel Therapeutics presented preclinical data for its investigational small‑molecule PX578 at the 2026 MDA Clinical and Scientific Conference. The drug is designed to activate the mitochondrial DNA polymerase gamma (POLG) and restore mitochondrial DNA levels in patients with mitochondrial DNA...
The FDA released draft Q&A guidance that clarifies how drug sponsors can qualify for three‑year non‑patent exclusivity by conducting a “new clinical investigation.” It defines a new study as a human, non‑bioavailability trial whose results have not been previously relied...
Friedreich ataxia (FA) received its first FDA‑approved therapy in 2023 when omaveloxolone, marketed as Skyclarys, earned accelerated approval. Biogen’s $7.3 billion acquisition of Reata Pharmaceuticals secured the drug and highlighted the market’s appetite for rare‑disease assets. Skyclarys works by activating the...
The study introduces a quantum‑annealing algorithm that predicts effective drug combinations by casting the problem as a quadratic unconstrained binary optimisation (QUBO). It leverages the network‑medicine concept of disease modules and the “Complementary Exposure” principle, which seeks drugs that hit...
The FDA has issued a safety alert warning consumers not to purchase or use Kian Pee Wan, a product marketed as an appetite stimulant and weight‑gain aid. Laboratory testing revealed the supplement contains the prescription steroid dexamethasone and the antihistamine...
CMS announced that the third round of the Medicare Drug Price Negotiation Program will proceed with full manufacturer participation, covering 15 high‑cost, single‑source drugs and one drug slated for renegotiation. The agency will issue initial price offers by June 1, 2026,...
Rates of Influenza and Pneumococcal Vaccination and Correlation with Survival in Multiple Myeloma Patients [Dec 6, 2022] @mtmdphd et al. @AjaiChari CLML https://t.co/kUQeRmdKWV #NCT02761187 #mmsm #IDonc #ClinicalTrials #caxtx https://t.co/L7r9caCcGN
MT @hhashmi87 #IMS25 Great Meet The Expert talk by @bdermanmd on MRD in Myeloma Risk vs benefit Who can stop treatment Active surveillance post discontinuation #mmMRD #mmsm https://t.co/YOBOPs7hQL
The FDA on March 10 expanded leucovorin’s label to cover a very rare genetic folate‑transport disorder, not autism. Earlier, Commissioner Marty Makary suggested the drug could help “hundreds of thousands of kids” with autism, creating expectations of a broad approval. FactCheck.org...
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/W3vwHRTzhE
Dompe, a privately owned biopharma, is advancing an intranasal nerve growth factor (NGF) therapy to treat non‑arteritic anterior ischemic optic neuropathy (NAION), a leading cause of sudden vision loss affecting roughly 6,000 U.S. patients annually. The approach leverages NGF’s neuroprotective...
Clinician attitudes and practices toward measurable residual disease in multiple myeloma [Jun 7, 2020] @bdermanmd @jagoda_jasielec @ajjakubowiak Brit J Haematol https://t.co/8zhxYfYwZQ #mmsm #mmMRD https://t.co/QTlR2gv5Q0
#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/IGDrNALHBP
An international research team has engineered a bacterial strain that boosts doxorubicin output by 180% compared with current industrial methods, overcoming three long‑standing bottlenecks—insufficient redox partners, drug‑binding “sponge” proteins, and suboptimal enzyme positioning. The findings, published in Nature Communications, detail...
SpyGlass Pharma’s Bimatoprost Drug Pad‑IOL System demonstrated significant intra‑ocular pressure (IOP) reductions and vision gains in a phase 1/2 trial of 104 patients with open‑angle glaucoma or ocular hypertension undergoing cataract surgery. At 12 months, the 78 µg dose lowered mean IOP 34%...
The U.S. Food and Drug Administration has approved Novartis' secukinumab, marketed as Cosentyx, for adolescents aged 12 and older with moderate‑to‑severe hidradenitis suppurativa (HS). This marks the first IL‑17A inhibitor cleared for a pediatric indication, extending the drug’s adult HS...
Kenai, a new allogeneic cell‑therapy company, is developing off‑the‑shelf iPSC‑derived progenitor cells for Parkinson’s disease, paired with a precision brain‑delivery platform that uses real‑time functional imaging. The approach builds on Japan’s recent approvals of iPSC‑based Parkinson’s treatments and aims to...
Researchers at Jeonbuk National University have unveiled DDINet, a lightweight neural network designed to predict drug‑drug interactions (DDIs) for previously unseen compounds. The model employs five fully‑connected layers and molecular fingerprints, with Morgan fingerprints delivering the best results. Using a...
A West Health‑Gallup survey reveals that over 82 million Americans have reduced daily spending to afford healthcare, highlighting deepening affordability pressures across income levels. The FDA has broadened GSK’s RSV vaccine Arexvy to cover high‑risk adults aged 18‑49, a group responsible...
The FDA has released a searchable table of extended use dates for specific drug lot numbers, based on manufacturer‑provided stability data. These extensions allow providers and patients to continue using existing stock beyond original expiration dates, helping to mitigate ongoing...
Recent peer‑reviewed study of 20 adults with chronic pain and substance misuse found ketamine therapy improved pain, mood, and dependence scores. The integrated treatment was delivered within a coordinated pain program, highlighting benefits of interdisciplinary care. Findings suggest ketamine can...
Researchers at INSERM uncovered hypothalamic tanycytes as a previously unknown tau‑clearing pathway, opening fresh therapeutic avenues for Alzheimer’s and related tauopathies. Merck Sharp & Dohme (MSD) announced the synthesis and pre‑clinical testing of novel PET tracers that bind α‑synuclein, aiming...
Shilpa Biologicals and mAbTree Biologics received FDA orphan drug designation for an investigational monoclonal antibody that targets an immune‑evasion pathway in essential thrombocythemia and polycythemia vera. The designation validates the novel mechanism and accelerates plans for IND‑enabling studies and first‑in‑human...
The FDA’s Center for Drug Evaluation and Research has expanded its Drug Safety Podcast series, delivering concise audio briefings that coincide with official Drug Safety Communications. Recent episodes address serious liver injury linked to Ocaliva, rare hypocalcemia risks with Prolia,...
The FDA released draft guidance that lets biosimilar developers use foreign comparator data and, in some cases, replace clinical studies with chemical analysis, potentially cutting development costs by $20 million. Simultaneously, the agency launched the Adverse Event Monitoring System (AEMS), a...
With FDA go ahead, a China #biotech notches a first in cell therapy testing https://t.co/QorwJmou8w by @realJacobBell $XENE $BHVN
The J. Craig Venter Institute unveiled a 4D, nanoscale simulation that tracks the entire life cycle of a minimal bacterial cell, marking a milestone for synthetic biology. A new gene‑editing platform designs DNA donors that dodge immune detection, enabling safer,...
San Francisco biotech Tahoe Therapeutics is building the world’s largest single‑cell atlas of cell‑chemical interactions, leveraging Integra Biosciences’ Assist Plus pipetting robot, Parse Biosciences’ Evercode scRNA‑seq kits, and its own AI analytics. Automation has increased single‑cell preparation throughput more than fivefold...
Cancer vaccines are showing marked efficacy vs refractory cancers and are going to be part of the future Rx armamentarium. Beyond that, ultimately, for prevention in high-risk individuals. A new stellar review @NatureMedicine https://t.co/PhtdZDtSBl https://t.co/okmwCD8AyB
The latest ASCO GU strategic intelligence report spotlights renal cell carcinoma (RCC) as a field entering a transformative phase. Analysts highlight emerging biomarkers, novel HIF‑2α inhibitors, and evolving immunotherapy combinations as potential high‑impact developments. While these advances promise to reshape...
100 brain microhemorrhages in a patient with cerebral amyloid angiopathy and 2 copies of APOE4 after amyloid antibody therapy https://t.co/ASpVuijFNW https://t.co/hdtwtv1usq
A new JAMA Internal Medicine study finds that the Inflation Reduction Act’s 2024 drug‑pricing provisions have reduced cost‑related medication nonadherence among Medicare beneficiaries. Compared with privately insured peers, seniors saw a 4.9‑percentage‑point drop overall and a 7.8‑point decline among those...
Gubra, a 300‑employee Danish biotech known for its obesity drug partnerships, announced plans to adopt a Roivant‑style incubation model. The strategy will spin out a series of focused biotech companies, each targeting niche therapeutic areas beyond obesity, such as metabolic...
Researchers have developed a self‑amplifying RNA injection that directs skeletal muscle to produce the heart‑healing peptide Nppa, dramatically reducing scar formation in pig models of myocardial infarction. The lipid‑nanoparticle‑delivered RNA sustains protein expression for at least four weeks, far outlasting...
NEW: Another failure in the clinic in the world of LAG-3, a target once seen as possibly the next major checkpoint inhibitor after PD-1 and CTLA-4. My latest on Immutep's Phase 3 disappointment, as LAG-3 focus now shifts to a...
