UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.
The funding comes weeks after TL1A blocker duvakitug maintained clinical remission rates above 50% in patients with ulcerative colitis and Crohn’s disease in a Phase 2b trial.
While an anonymous source tied the closure to shortcomings in the FDA’s new pathway, a spokesperson for the Department of Health and Human Services pushed back on the suggestion.
Sanofi will gain global exclusive rights over rovadicitinib, an oral JAK/ROCK blocker that has anti-inflammatory and anti-fibrotic effects.
Follow along as BioSpace tracks job cuts and restructuring initiatives.
With fresh billions unlocked in the 2026 U.S. budget and mission‑driven family offices recalibrating after a “nuclear winter,” early stage biotechs are rewriting their financing strategies around nondilutive capital and targeted private wealth.
FEATURED STORIES
Poplar Therapeutics is seeking a “step change” in the treatment of food allergy and other atopic conditions, with $95 million raised to date, including a $45 million series A extension that closed Tuesday.
Infrastructure and location have helped make Holly Springs a future hub for obesity drug production, with Amgen and Roche planning to manufacture GLP-1 therapies there to compete in the growing market.
Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies.
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking.
Generate:Biomedicines has hit the public markets as the world begins to question the usefulness of AI technology. CEO Mike Nally says biology is the key to unlocking the technology’s full potential.
The CDC’s changes threaten to cut vaccine sales for makers including Pfizer, Moderna, Merck and more, but a legal expert suspects affected manufacturers will stay on the sidelines rather than back a push to declare the revised schedule unlawful.
FROM BIOSPACE INSIGHTS
The U.S. Congress greenlit a historic $315 million in federal ALS research funding for 2026 amid Rare Disease Month, spotlighting biotech progress like VectorY Therapeutics’ first patient dosing in its TDP-43-targeting PIONEER-ALS trial and EverythingALS’ pharma consortia driving biomarker innovations and trial alignment.
LATEST PODCASTS
Amylyx looks to the future after Relyvrio withdrawal, ADCs continue to attract investment and the drug shortage persists in the U.S.
While women’s clinical trial participation has increased, clinical trials still largely are not designed for women. In this episode, we discuss the many areas where trials can be improved, such as human and historical diagnosis and screening biases, accessibility, data capturing, and more
Plus, how the geopolitical tensions with China will affect U.S. biopharma
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SPECIAL EDITIONS
The BioSpace team hit the ground running at the J.P. Morgan Healthcare Conference earlier this month to bring you the news from the streets of San Francisco.
BioSpace data show job postings live increased quarter over quarter, while layoffs fell year over year.
Recent breakthroughs and three decades of progress in treating Huntington’s disease
DEALS
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While Boehringer Ingelheim hasn’t yet revealed what diseases it will go after, Sitryx’s oral drug candidate could potentially be disease-modifying for a variety of autoimmune and inflammatory conditions. -
Pfizer will be responsible for all global clinical and regulatory activities for Beam Therapeutics’ liver-directed gene editor. -
After its next-generation obesity asset CagriSema lost a head-to-head matchup with Eli Lilly’s Zepbound, Novo Nordisk is throwing more money into the obesity space, striking a deal with Vivtex to advance novel weight loss pills. -
Ecnoglutide, which Pfizer licensed from Sciwind Biosciences, is already approved in China for type 2 diabetes mellitus, and a marketing application for weight loss has been accepted by regulatory authorities in the country. -
In a Phase 1 study, 82% of patients on VIR-5500 achieved at least a 50% reduction in PSA levels—a result analysts praised as competitive in the prostate cancer space.
WEIGHT LOSS
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As Novo Nordisk continues to lose ground in the obesity market to rival Eli Lilly, the Danish company has started construction projects to establish the ex-Alkermes plant as a hub for supplying oral GLP-1 products to global markets. -
On the FDA’s docket this month are two decisions pushed back from 2025, including one for a rare form of obesity and another for dry eye disease. -
Novo Nordisk reported a loss in a head-to-head trial of CagriSema against Lilly’s Zepbound earlier this week. This time around, Lilly’s orforglipron bested Novo’s oral semaglutide in blood sugar control and weight reduction—albeit with a few extra discontinuations as compared to its rival. -
Slate Medicines will move forward with a migraine drug from a Chinese biotech, while Alveus Therapeutics will advance a dual GLP-1/GIPR fusion protein for weight loss. -
Eli Lilly notches another win over Novo Nordisk, as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another leadership change shakes up CDC; and Gilead acquires CAR T partner Arcellx for nearly $8 billion.
POLICY
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One of the two new members of the CDC’s Advisory Committee on Immunization Practices questioned the safety of COVID-19 vaccines before the Texas Senate in 2021. -
This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments. -
FDA Commissioner Marty Makary presented a new idea to staff this week: bonus payments for employees that complete regulatory review processes faster than expected. -
A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency. -
Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.
With leaner teams and tighter budgets, senior leaders can face tremendous strain as they juggle increased workloads and leadership responsibilities. In this column, Kaye/Bassman’s Michael Pietrack discusses how pressure builds and what can ease it.
Biopharma professionals need to understand today’s job market and how they can stand out to position themselves for success. Three talent acquisition and recruiting experts discussed these topics in a BioSpace webinar, from the importance of contract work to the value of an advocate.
This webinar provides a clear-eyed assessment of current hiring conditions across biopharma, exploring which roles and skills are in demand, where opportunities are emerging, and how hiring practices are evolving.
Looking for a biopharma job? Check out the BioSpace list of 12 top companies hiring life sciences professionals like you.
If workloads aren’t adjusted as needed, the company’s priorities are already compromised. Executive coach Angela Justice explores what happens when goals move forward without removing unnecessary work and what to do about it.
At some point in your research career, you may find yourself transitioning from academia to industry or vice versa. To best set yourself up for success, adapt your approach to the specific scientific culture where you work.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with muscle-invasive bladder cancer, even those eligible for cisplatin treatment. -
Solstice Oncology will gain an exclusive worldwide license to Harbour BioMed’s porustobrt, an anti-CTLA-4 antibody currently being studied for melanoma, colorectal cancer and other malignancies in China. -
The necessity of delivering medicine days after it’s produced drives decisions about where to build facilities and how to ship radioactive materials to healthcare providers. -
The centerpiece of the takeover is anito-cel, a CAR T therapy under development for relapsed or refractory multiple myeloma. An FDA decision on the therapy is expected by December 2026. -
Suppliers are investing in production to support deals with AstraZeneca, Bayer and other drugmakers that are advancing radioisotope-based cancer therapies.
NEUROSCIENCE
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In this episode of Denatured, you’ll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We’ll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints. -
Bysanti is based on iloperidone, an active metabolite of a compound that forms the core of Fanapt, another drug by Vanda Pharmaceuticals. -
Tracy Beth Høeg addressed FDA staffers for the first time in her role as the fifth CDER chief under President Donald Trump, announcing inquiries into the use of SSRIs in pregnancy and RSV antibodies in infants despite well-documented safety of these treatments. -
Korsana’s lead program uses a next-generation shuttling technology to improve delivery into the brain and lower the incidence of amyloid-related imaging abnormalities. -
The FDA outright refuses to review Moderna’s mRNA-based flu vaccine as CBER director Vinay Prasad’s conduct is scrutinized; Disc Medicine receives an unexpected rejection, which Prasad may also have had a hand in; Compass Pathways posts new late-stage data on its psilocybin-based depression drug; CDC is once again leaderless.
CELL AND GENE THERAPY
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The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later. -
In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes. -
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval. -
After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline. -
The rejection of Atara Biotherapeutics’ Ebvallo in January was a “complete reversal” of the conesensus FDA reviewers had come to, according to a former agency employee, who said manufacturing problems were the only approvability barrier for the drug.
