DDW Highlights: 26 March 2026
In this episode, Bruno Quinney highlights three breakthrough studies: Edinburgh researchers engineered E. coli to convert PET plastic waste into the Parkinson's drug L‑DOPA, offering a sustainable route to a vital medication; scientists identified the enzyme DHX8 as a key regulator of the stress‑response protein HSF1 in cancer cells, revealing a new vulnerability for drug development; and new work pinpointed kappa and lambda myeloma antigens as highly specific targets for next‑generation immunotherapies in multiple myeloma. Each story underscores innovative bio‑upcycling, novel molecular targets, and precision medicine advances.
Otsuka Pharmaceutical Reports P-III (VISIONARY) Trial Data on Voyxact for IgA Nephropathy (IgAN)
Otsuka Pharmaceutical presented Phase III VISIONARY trial data for Voyxact (sibeprenlimab‑szsi) in IgA nephropathy patients at risk of progression. At 48 weeks, 82.5% of patients receiving 400 mg subcutaneous Voyxact achieved negative microscopic hematuria versus 52.6% on placebo, with median time to...
Anti‑TGFβ1 Antibody Shows Durable Efficacy, Low Toxicity
Scholar Rock's anti-TGFb1 antibody clinical study with early data in oncology indications... minimal AEs and signs of durable efficacy in checkpoint-refractory patients. $SRRK https://t.co/8xsN4wtbMV
Development of an Ultra-Sensitive Human Cardiac Troponin I Sandwich ELISA
Exazym®'s BOLD amplification technology boosts the sensitivity of a human cardiac troponin I sandwich ELISA by 180‑fold, lowering the detection limit to 0.07 pg/mL. The webinar presented by Cavidi’s Peter Stenlund shows how the method integrates into standard ELISA workflows with...
Autoimmune Immunotherapy Is Shifting Upstream: AnaptysBio on Targeting Pathogenic Immune Cells
Autoimmune drug development is moving upstream, targeting pathogenic immune cells rather than single cytokines. AnaptysBio’s Chief Medical Officer, Paul Lizzul, highlighted the company’s cell‑selective immunomodulation strategy, including CD122 antagonism that modulates both CD4 helper and CD8 cytotoxic T cells. Early‑phase...
Senate Advances Bill to Fund Psychedelic Trials for Veterans' Mental Health
The U.S. Senate has advanced legislation that would fund expanded clinical trials of psychedelic therapies for veterans suffering from PTSD, depression and traumatic brain injury. The bill, backed by a bipartisan coalition, marks the first federal effort to systematically study...
Eli Lilly’s Triple‑Agonist Retatrutide Cuts A1C 2% and Weight 17% in Phase III
Eli Lilly announced top‑line results from the Phase III TRANSCEND‑T2D‑1 trial, showing its investigational triple‑hormone agonist retatrutide reduced HbA1c by up to 2 percentage points and produced an average 16.8 % weight loss over 40 weeks. The data position retatrutide as a potential...
Atossa Therapeutics Posts Q4 2025 Results, Secures Rare Disease Designations for (Z)-Endoxifen
Atossa Therapeutics released its fourth‑quarter 2025 earnings and announced that the FDA granted Rare Pediatric Disease and Orphan Drug designations to its lead candidate (Z)-endoxifen for Duchenne muscular dystrophy. The company said the designations accelerate its rare‑disease strategy while it...
Prescribing CORT Lifyorli Differs From Cushing’s Experience
Here's prescribing info for $CORT Lifyorli -> https://t.co/i46Whl5lX6 Why so different from the experience in Cushing's? https://t.co/EqvbCApqHn
Dyadic COO Hazelton Steers Modest $3.1M Revenue Rise Amid Grant‑driven Cost Shift
Dyadic (DYAI) posted Q4 2025 revenue of $3.1 million, a modest rise driven by COO Joseph P. Hazelton’s operational focus. The earnings call highlighted new product launches, global distribution deals and a growing reliance on grant‑linked R&D, offsetting a drop in...
Corcept Therapeutics Stock Jumps 19.7% After FDA Approves Lifyorli Combo
Corcept Therapeutics saw its shares climb almost 20% after the U.S. FDA gave the green light to Lifyorli in combination with nab‑paclitaxel for platinum‑resistant ovarian, fallopian tube and primary peritoneal cancers. The approval, based on a 381‑patient Rosella trial, validates...
World-First Living ‘Robots’ Develop Functional Nervous Systems
Researchers at the Wyss Institute have created the first living robots, called neurobots, that develop functional nervous systems from implanted neuronal precursor cells. The neurobots, built from frog embryonic cells, self‑organize neural networks that reshape their morphology, boost motility, and...
Mini Brains Rewire to Balance Digital Pole
🔺 These Mini Brains Just Learned to Solve a Classic Engineering Problem 🧠 In a step toward biological computing, brain organoids rewired their networks as they learned to balance a digital pole on a cart. https://t.co/w2wHDTvIVp https://t.co/h51xTVlR4D
Biogen and Alteogen Partner for ALT-B4 to Advance Subcutaneous Formulations of Biologics
Biogen has signed an exclusive license with South Korean biotech Alteogen for ALT‑B4, a recombinant hyaluronidase enzyme that facilitates subcutaneous administration of biologics traditionally given intravenously. The agreement provides Alteogen with $20 million upfront, a $10 million payment upon initiation of a...
LPBF Prints Zinc–Silver–Copper Alloys For Biodegradable Implants
Researchers used laser powder bed fusion (LPBF) to 3D‑print zinc‑silver‑copper alloys and demonstrated in‑vitro cytocompatibility, indicating the material could serve as a biodegradable implant. Zinc offers a middle‑ground degradation rate between magnesium and iron, while silver and copper add antimicrobial...
Probiotics Drink Reduces Loose Stools Tendency in Healthy Adults - Study
A randomized, double‑blind trial in Japan found that a daily 100 ml drink containing 10 billion CFU of Lactiplantibacillus plantarum 299v reduced bowel‑movement frequency and days with defecation in healthy adults prone to loose stools. After eight weeks, the probiotic group averaged 6.7...
ELMED Life Sciences Raises $2.7 Mn in Series A From NABVENTURES
ELMED Life Sciences secured a $2.7 million Series A round led by NABVENTURES' AgriSURE Fund. The capital will fund new manufacturing capacity in Hyderabad, R&D acceleration, and broader distribution across Indian tier‑2/3 regions and overseas markets. ELMED develops probiotic and microbiome‑based products...
Beyond the Cornfields, Indiana’s Life Sciences Industry Grows Tall
Indiana’s life‑sciences sector is rapidly expanding, now encompassing over 3,300 companies that employ roughly 70,000 workers and generate a $102 billion economic impact. Flagship firms such as Eli Lilly are driving major investments, including a $13 billion Medicine Foundry slated to create 400...
China’s Jiangsu Aidea Eyes Hong Kong Listing as Gateway to US and European Aids Markets
Jiangsu Aidea Pharmaceutical, a Shanghai‑Star Market listed firm specializing in HIV/AIDS therapies, is preparing a Hong Kong IPO to fund its expansion into the United States and Europe. The company projects the global AIDS‑treatment market to exceed $1.45 billion by 2027...
Lenz Therapeutics Shares Drop 11% After Q4 Earnings Miss
Lenz Therapeutics' shares slid 11.2% after the biotech reported a Q4 loss of $1.16 per share on $1.6 million in revenue, well below analyst expectations. The miss stemmed from weaker-than-anticipated uptake of its newly launched VIZZ eye‑drop and a surge in...
Palvella Director Buys $500K After Phase‑3 Win, COO Sells Options
Palvella Therapeutics director George M. Jenkins invested $500,000 in the company by buying 4,000 shares at $125 each during a public offering that closed after the firm announced a Phase‑3 trial win. At the same time, chief operating officer Kathleen...
LEV Foundation Partners with Human Longevity Inc.
Delighted to share that LEV Foundation is initiating a collaboration with Human Longevity Inc. - details here: https://t.co/Ipfud85sGG
Hong Kong: Research and Tech Driving Life and Health Innovation
Hong Kong Science and Technology Parks Corporation convened leading life‑science firms, investors and partners at its CTC Marketplace to showcase the city’s emerging health‑tech ecosystem. The event highlighted Hong Kong’s strengths in translational research, clinical‑trial capacity and AI‑driven drug discovery,...
MHRA’s Tallon on the Tall Order of Boosting the U.K. as a Destination for Clinical Trials
Lawrence Tallon, the MHRA chief, is set to oversee the launch of the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025 on April 28, 2026. The amendment represents the most extensive overhaul of U.K. clinical‑trial rules in two decades, targeting faster approval,...
Passion Fruit Compound Shields Mitochondria, Improves Mouse Memory
Alpha-amyrin, a molecule found in passion fruit, has demonstrated the ability to protect brain mitochondria and reduce memory loss in Alzheimer's mouse models, suggesting potential for future therapeutic development. neuroscience
Restarting SSRIs Improves Outcomes Over Med‑Free Approach
When we published our H2H in @NEJM Ian Jordan raised an excellent letter re SSRI discontinuation & how it might impact response. We looked & found he was right. Those who discontinued SSRIs and went back on (escitalopram) did far...
Regulatory Education for Industry (REdI) Annual Conference 2026: Innovative Regulatory Strategies to Advance Medical Products - 05/19/2026
The FDA’s Regulatory Education for Industry (REdI) Annual Conference will take place May 19‑20, 2026, offering both virtual and in‑person sessions at the White Oak Campus in Maryland. The two‑day program features three dedicated tracks—drugs, devices, and biologics—allowing participants to...
FDA Approves Avlayah as Treatment of Hunter Syndrome
Denali Therapeutics received accelerated FDA approval for Avlayon (tividenofusp alfa‑eknm), the first enzyme replacement therapy designed to cross the blood‑brain barrier for Hunter syndrome. The approval is based on a phase 1/2 trial of 47 boys up to 18 years, showing...
FY 2025 GDUFA Science and Research Report
The FDA’s Center for Drug Evaluation and Research released its FY 2025 GDUFA Science & Research Report, detailing more than 50 funded projects across eight priority scientific initiatives. The program targets bioequivalence, manufacturing standards, and advanced analytical methods to streamline abbreviated...
Patent Certifications and Suitability Petitions
Under the Hatch‑Waxman law, generic manufacturers must file a Paragraph IV certification asserting that a listed patent is invalid, unenforceable, or not infringed to obtain FDA approval. The first substantially complete ANDA with such a certification secures a 180‑day exclusivity period,...
This Cutting-Edge Treatment Hit the Rewind Button On Aging, Scientists Say
Researchers at Longeveron reported that a single infusion of laromestrocel, a mesenchymal stem‑cell therapy derived from donors aged 14‑18, significantly boosted mobility in frail seniors. In a double‑blind trial of about 150 participants aged 70‑85, the highest dose (200 million cells)...
IP Considerations Following FDA Announcement on Flexibility for Cell and Gene Therapies
The FDA announced new guidance that expands flexibility in chemistry, manufacturing, and controls (CMC) for cell and gene therapies (CGTs) across their development lifecycle. Sponsors can now defer full cGMP compliance until later trial phases and make iterative manufacturing changes...
FDA Approves Drug to Treat Neurologic Manifestations of Hunter Syndrome
The FDA granted accelerated approval to Avlayah (tividenofusp alfa‑eknm), a weekly IV infusion, for treating neurologic manifestations of Hunter syndrome in pediatric patients weighing at least 5 kg. The approval is based on a phase 1/2 trial that demonstrated a 91% average...
Leveraging the Full Potential of Regenerative Medicine Requires a Proactive Approach
Regenerative medicine promises to shift healthcare from a reactive model to proactive disease modification by targeting early biological drivers of chronic degeneration. Cell‑based therapies such as mesenchymal stromal/stem cells (MSCs) can modulate inflammation, immune signaling, and tissue repair, showing benefits...
Parasites Prompt Gut-Brain Communication to Trigger Appetite Loss
UCSF researchers have mapped a gut‑brain signaling cascade that explains why parasitic worm infections cause loss of appetite. They discovered that tuft cells detect parasite‑derived succinate and release acetylcholine, which prompts nearby enterochromaffin cells to secrete serotonin. The serotonin then...
Psilocybin Treatments for Treatment-Resistant Depression with Compass Pathways’ Dr. Steve Levine — Episode 248
The Xtalks Life Science Podcast featured Dr. Steve Levine, Chief Patient Officer at Compass Pathways, discussing the company’s push to develop psilocybin‑based therapies for treatment‑resistant depression (TRD). Levine, a board‑certified psychiatrist and founder of Actify Neurotherapies, highlighted the clinical promise...
Pharmaceutical Executive Daily: FDA Issues Warning Letter to ImmunityBio
The FDA issued a warning letter to ImmunityBio for misleading promotional claims about its bladder‑cancer drug Anktiva, triggering a roughly 26 percent drop in the company’s shares and giving it 15 days to submit a corrective plan. In parallel, Merck announced a...
GlycoNet – Sugar-Based Vaccine Against Bacterial Diarrhea Shows Promise in Phase 1 Trial
Researchers at the University of Guelph announced that their sugar‑based vaccine candidate against Campylobacter jejuni demonstrated safety and immunogenicity in a small Phase 1 human trial. Participants experienced only mild side effects, and the formulation generated measurable antibody responses even at...
Open-Source DIY Guide to Building mRNA Vaccines
Inspired by the man who built a personalized cancer vaccine for his dog, I’ve written an open-source guide to DIY mRNA vaccine production: philfung.github.io/openvaxx Drawing on my background in running lab startups, the guide covers the entire process - from sequencing...
Anavex Sinks After Pulling Alzheimer's Filing in EU
Anavex Life Sciences withdrew its European marketing authorisation application for blarcamesine, an add‑on therapy for early‑stage Alzheimer’s, after the EMA’s CHMP signaled a likely negative opinion. The committee criticized the trial’s efficacy data, methodological flaws, and safety reporting, including concerns...
Drug Quality Sampling and Testing Programs
The FDA’s Center for Drug Evaluation and Research (CDER) released its FY 25 Drug Quality Sampling and Testing results, showing that the vast majority of tested pharmaceuticals met USP specifications. The program uses a risk‑based, data‑driven approach introduced in 2018 to...
Aging Impairs Activation of Muscle Stem Cells, with MG53 as a Potential Target for Therapies
Researchers have shown that age‑related muscle loss stems primarily from a decline in the activation of resident muscle stem cells, not from their depletion. Early activation of these satellite cells is a stress‑sensitive, rate‑limiting step that becomes impaired in older...
Summit Sparks Breakthroughs: AI, Gene Therapy, CRISPR, mRNA
One thing that really makes the in-person summits we run at STAT so amazing: the people in the room. Here's what happened when we asked some of them for the last big breakthrough they saw. Featuring: the infectious @DrBlytheAdamson, genomcis pioneer...
First Full GlyphAllo Data Reveal From Discovery to Human Proof‑of‑Concept
Proud of the work of our Seaport team & collaborators published today in @ScienceTM. This ~13 page peer reviewed paper is the first comprehensive data disclosure of our GlyphAllo™ program from discovery through initial human proof‑of‑concept.
Microscopic Robot Delivers Targeted Drugs Inside Bloodstream
Microscopic #Robot Navigates the Bloodstream to Deliver Targeted Medication by @CeoImed #MedTech #Healthcare #HealthTech #Tech #Technology https://t.co/LBJeDdwTuq
Open Review Flags Flaws in Telomere‑river Longevity Study
The incredible study using "Rivers of telomeres" to rejuvenate tissues and extend lifespan in mice is now undergoing open peer-review. I did a review, pointing out issues with the methods and the lifespan curves. They're looking for more reviewers. https://t.co/KOiR4F85sT
Blueprint for Affordable, Accessible AAV Gene Therapy
When people ask "How can we make #AAV #GeneTherapy accessible and affordable" --> this is how 👇
New Review Links Β‑cell Stress to Type 1 Diabetes
Rethinking why the pancreas β-cells become vulnerable to autoimmune attack, leading to Type 1 diabetes. An illuminating review with ideas to counter β-cell stress @ScienceTM https://t.co/zn6a6wEg7u https://t.co/BV0LYnWRNL