
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

GH Research’s inhaled mebufotenin (GH001) demonstrated ultra‑rapid, profound antidepressant effects in a Phase 2b trial for treatment‑resistant depression, with 73% of patients in remission after six months versus ~11% for standard oral therapies. Early‑stage studies in postpartum depression and bipolar disorder showed similarly swift remission, including a ten‑patient postpartum cohort where all achieved remission. GH001’s inhaled delivery yields onset within seconds and a brief 11‑minute psychoactive window, enabling same‑day discharge and potential integration into existing Spravato‑style clinic networks. The company is now preparing a pivotal Phase 3 program while remaining well‑capitalized and publicly traded.

Goal-Driven Flexible Bayesian Design presentation updated w/comparison of performance of frequentist group sequential designs: https://t.co/QCZ6DQrm07 . Frequentist approach takes far too long to make a decision by controlling something that is NOT an error prob. #Statistics #rct https://t.co/X2knbmzTuX
My @CNBC segment on how President Trump's drug pricing deals will achieve many of the initial aims set out by tariff proposals, with respect to reshoring drug manufacturing, and how the biggest consumer impact in terms of lower drug costs...
Gilead Sciences agreed to acquire the remaining shares of Arcellx for an equity value of $7.8 billion, paying $115 per share—a 79.4% premium to the prior close. The deal secures full rights to Arcellx’s BCMA‑directed CAR‑T therapy anito‑cel, which is in...
Novo Nordisk’s experimental obesity drug CagriSema delivered a 23% average weight loss in the 84‑week REDEFINE 4 Phase 3 trial, but fell short of Eli Lilly’s tirzepatide (Zepbound) which achieved 25.5%. The head‑to‑head study failed the primary non‑inferiority endpoint, prompting a 15% drop...

Gossamer Bio announced that its Phase 3 trial of seralutinib for pulmonary arterial hypertension missed its primary endpoint. Patients on seralutinib walked 13 meters farther than placebo over six minutes, but the difference lacked statistical significance. The trial also reported cough...

Evaluating a doctor-in-the-loop AI-agent for multiple myeloma chart reviews [Nov 3, 2025] Karla Mariana Castro Bórquez et al. @HealthTree #ASH25 abs25-15262 PID 4424 https://t.co/MvBeHqOxCg #mmsm #AI #EMR #RWD https://t.co/onY817NfVr

Individualized treatment risk stratification using histopathology-based genomics prediction in multiple myeloma: A multicenter Study in 1429 participants [Nov 3, 2025] Rajanna et al. @DrOlaLandgren #ASH25 abs25-11635 PID 4008 https://t.co/gOprMCjfgh #mmsm #AI #oncopath https://t.co/pjikEsgfHC
At the BIO Investor Growth (BIG) Summit, IBD Ventures’ associate director Nicole Schwerbrock discussed how venture philanthropy and patient advocacy are reshaping investment in inflammatory bowel disease (IBD) research. She explained that patient‑driven funding models complement traditional venture capital by...

Race agnostic models maintain accuracy in predicting M-protein levels in multiple myeloma [Nov 3, 2025] @rahul2u et al. #ASH25 abs25-11347 PID 2810 https://t.co/A27ozBoW8h #mmsm #AI #CancerDisparities https://t.co/ubJ6NGMFUE

Unstructured serum electrophoresis data limits real world AI models in multiple myeloma: Evaluation of 384 institutions across United States [Nov 3, 2025] @MalekEhsanMD et al. #ASH25 abs25-11278 PID 2809 https://t.co/mAC8l7xnaG #mmsm #cancerdisparities #hpeonc https://t.co/veJxBV5S0N
Researchers identified oligodendrocyte myelin glycoprotein (OMG) in blood as a marker inversely associated with cortical amyloid‑β deposition and neurodegeneration. Large‑scale plasma proteomics across more than a dozen cohorts showed lower OMG levels in individuals with Alzheimer’s disease, other dementias, and...

Patient perspectives & preferences for step-up dosing and treatment w/ teclistamab & talquetamab: Insights from a patient survey [Nov 3, 2025] @JayHydren et al. @RahulBanerjeeMD abs25-8335 PID 6354 https://t.co/pRlflaSb2I #mmsm #Tcellrx @HealthTree https://t.co/3I9nwsxZMx
The HealthTree Cure Hub registry: A patient-centered, multicenter approach to advancing multiple myeloma care - Jorge Arturo Hurtado Martínez et al. @HealthTree #ASCO25 Abst e19569 https://t.co/X2pdaIDYth #mmsm

Gilead Sciences announced a $7.8 billion acquisition of Arcellx, pricing the deal at $115 per share—a 79% premium to the prior close. The agreement includes an additional $5 per share contingent on future sales milestones. Central to the transaction is anito‑cel,...
Patient Preferences on Clinical Decision-Making in Myeloma: A 747-Patient Survey [Dec 7, 2024] @ManniMD1 et al. - @HealthTree https://t.co/jATJRKEfB2 #ASH24 Abst 2281 #mmsm HT @Taxkourel
Patient-Centered Care in Hematology: How Side Effect Severity Influences Decision-Making Autonomy in Relapsed Refractory Multiple Myeloma [Dec 8, 2024] Martinez et al. #ASH24 Abst 706 https://t.co/l7SHPBwbna #mmsm HT @Taxkourel

Jay Bregman, CEO of Andel, says the FDA’s pending crackdown on non‑FDA‑approved GLP‑1 compounds is long overdue and will target the burgeoning market of compounded semaglutide. He estimates roughly 1.5 million patients currently rely on these unapproved products, a figure he...
The effect of care team-patient relationship on decision-making in relapsed/refractory multiple myeloma - @MyelomaTeacher et al. @HealthTree #ASCO24 Abstract e19530 https://t.co/Ss5LZHH0Oj #mmsm
A survey on the patient perspective on cure in multiple myeloma [Oct 9, 2022] @ManniMD1 et al. @HealthtreeMM @TheLancetHaem https://t.co/1XhKT7AWGi #mmsm

BioNTech has filed a Delaware patent‑infringement lawsuit against Moderna, accusing the latter’s next‑generation COVID vaccine mNEXSPIKE of using BioNTech’s streamlined mRNA design. The disputed shot is projected to generate about 55 % of Moderna’s COVID revenue this season, making the case...

.@HealthtreeMM Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles & Accelerate Research in Multiple Myeloma [3/10/22] @NSweeneyPhd @AhlstromJenny @FaithEDavies1 @mtmdphd @JCOCCI_ASCO https://t.co/WOwLb9Eozv @myelomacrowd #mmsm https://t.co/dZzhU3N32Q

. @HealthtreeMM Patient Portal mediated myeloma patient reported diagnostic imaging and pathology testing - @NSweeneyPhd @AhlstromJenny @mtmdphd J Clin Oncol 38: 2020 (suppl; abstr e20565) #ASCO20 https://t.co/CRLXETpeMm #mmsm #oncorad #oncopath https://t.co/m1UGDimLWm
Partner Therapeutics presented post‑hoc data from its Phase II eNRGy study of zenocutuzumab (Bizengri) in 27 patients with advanced NRG1‑positive non‑small cell lung cancer who received at least three doses beyond radiographic progression. Eight patients stayed on therapy for six months...

Lenalidomide + high-dose dexamethasone (RD) vs lenalidomide + low-dose dexamethasone (Rd) as initial therapy for newly diagnosed multiple myeloma: an open-label RCT [10/22/2009] @VincentRK et al. @TheLancetOncol https://t.co/hXxlFINejC #NCT00098475 #EAonc E4A03 #mmsm #caxtx #ctsm https://t.co/FjBnt6yIox

#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/QdWETmSoiR

Novo Nordisk’s next‑generation obesity drug CagriSema failed to demonstrate non‑inferiority to Eli Lilly’s tirzepatide in the 84‑week REDEFINE 4 study, achieving 20.2% weight loss versus 23.6% for tirzepatide. The open‑label trial showed the two treatments were not statistically equivalent. Following the announcement,...

EAA241 - Ph 2 RCT Dara-Bor-Dex vs Cy-Bor-Dex in the treatment of Newly Diagnosed Multiple Myeloma with Light Chain Cast Nephropathy (LCCN) [Study activated 8/11/25] @keruakous https://t.co/1NgvVZ3fTA #NCT07085728 #mmsm @eaonc https://t.co/paGRlHV1Dv
Johnson & Johnson released long‑term QUASAR extension data for Tremfya (guselkumab) in ulcerative colitis, showing sustained efficacy through week 140. Clinical remission was achieved by 80.8% of patients, with 78.6% attaining histo‑endoscopic improvement and 53.6% reaching endoscopic remission. Approximately 89% of...
The 2025 earnings season highlighted a clear hierarchy among healthcare giants, with Eli Lilly and AstraZeneca leading pharma revenue growth and Medtronic and Edwards Lifesciences topping medtech sales. The podcast hosted by Robert Barrie and Ross Law breaks down the financial results, noting...

Health Secretary Robert F. Kennedy Jr. has pledged to purge industry influence from the FDA, yet the Trump administration’s injection of political priorities has reshaped the agency’s operating environment. Under the current administration, discussions that once were deemed off‑limits between...

Vanda Pharmaceuticals announced that the U.S. FDA has approved Bysanti (milsaperidone), an active metabolite of iloperidone, as a first‑line therapy for acute manic or mixed episodes in bipolar I disorder and for schizophrenia. The approval is based on demonstrated bioequivalence...

The FDA, backed by HHS, issued a stark warning to compounding pharmacies that market GLP‑1 products such as semaglutide, after Hims & Hers promoted a compounded Wegovy copy. A press release announced intent to restrict non‑approved GLP‑1 APIs and referred...

Emma Dyer bought weight‑loss injections online for £115, bypassing any medical assessment, and suffered a severe health collapse within days. Her experience reflects a wider surge, with an estimated 1.6 million UK adults using GLP‑1 drugs such as Mounjaro and Wegovy,...
Summit Therapeutics reported a strong cash position of $713 million with no debt at the end of 2025, while GAAP operating expenses fell to $225 million despite a rise in non‑GAAP R&D spend. The FDA accepted the Biologics License Application for ivonesumab...
Tarsus Pharmaceuticals reported record 2025 net product sales of $451.4 million, driven by XDEMVY adoption, and issued its first full‑year 2026 guidance forecasting $670‑$700 million, implying over 50% revenue growth. The company highlighted a 44% gross‑to‑net discount, a robust DTC campaign that...
AbbVie reported a record $61.2 billion in net revenue for 2025, an 8.6% increase that beat guidance despite a $16 billion HUMIRA erosion. Adjusted EPS reached $10, $0.54 above the midpoint, while SKYRIZI and RINVOQ together generated $7.4 billion in Q4, delivering double‑digit...
Axsome Therapeutics reported a 66% revenue surge to $639 million in 2025, driven by strong growth in Auvelity, Sunosi and the newly launched Cymbravo. Net loss narrowed to $183 million, a 35% improvement year‑over‑year, while cash rose to $323 million. The company secured...
BioMarin Pharmaceutical raised its fiscal 2025 total revenue guidance, now starting at $3.15 billion, and reaffirmed Voxzogo revenue between $900 million and $935 million. The company lifted non‑GAAP operating margin guidance to 26‑27% and lifted diluted EPS guidance to $3.50‑$3.60. A $221 million IPR&D...

Japan’s health ministry is set to grant conditional approval to two first‑of‑a‑kind regenerative medicines – Amchepry for Parkinson’s disease and ReHeart for severe heart failure – after tiny phase I/II trials involving seven and eight patients respectively. Both drugs are derived...
Novavax (unlike the mRNA vaccines) generates antibodies to both S1 and S2 units of the spike protein - explaining why it provides somewhat broader protection. I've personally gotten Novavax every time it was available at vaccine time.
22February: What does @ForesiteCapital look for in the therapeutics and instrument startups in which it invests? Read founder & CEO @JTananbaum MD's response + more insights from him and the firm's Michael Rome PhD in my latest Q&A interview for...

The complexity of our immune system is daunting. But there's a path to deconvolute it and understand causal relationships. It's CRISPR. open-access @JExpMed https://t.co/QF6Cvkbfz2 https://t.co/OTL2z1fow0
The key point here is that the proposed study would not have equipoise because almost certainly some babies in the control arm would get hepatitis from their infected mother.
Submitting an abstract for a late-stage large clinical trial for review at a major conference, where you're asking for an oral, but only having 2 authors should be disqualifying. Really, only *2* people acquired ALL the data for this? Disgraceful...
Teclistamab–Daratumumab in Relapsed or Refractory Multiple Myeloma [Feb 18, 2026] @End_myeloma et al. @NEJM https://t.co/PTSmdrV2zp #mmsm #tcellrx #ImmunoOnc QUICK TAKES: New phase 3 trial results are summarized in a short video.
Exposure–Response Analyses for Therapeutic Dose Selection of Belantamab Mafodotin in Patients with Relapsed/Refractory Multiple Myeloma [Sep 1, 2021] Ferron-Brady et al. Clinical Pharmacology & Therapeutics https://t.co/58kmDFKRwO #mmsm #oncopharm #caxtx
Targeting BCMA in Multiple Myeloma [Aug 25, 2021] Tan & @UrviShahMD Current Hematologic Malignancy Reports https://t.co/lAhqwwa7u3 #mmsm #caxtx #CARTcell

Immunoglobulin Isotype Switch after Anti-BCMA CAR T-Cell Therapy for Relapsed or Refractory Multiple Myeloma [Nov 10, 2021] Liang et al. @BloodAdvances https://t.co/KfreDCvIDH #CARTcell #mmsm https://t.co/7qNQBEoqkX

Antigen escape as a shared mechanism of resistance to BCMA-directed therapies in multiple myeloma [Jul 25, 2024] @RossFirestone et al. @BloodJournal https://t.co/cOh0YC2u9H #mmsm #ImmunoOnc #CARTcell #PrecisionMedicine #lymphoidneoplasia #immunobiologyandimmunotherapy https://t.co/6sbEv5KtLY