The move comes as BioNTech shifts to being a multiproduct commercial biotech, allowing Ugur Sahin and Özlem Türeci to transition back into research on next-generation mRNA therapeutics.
Analysts expect the market for manufacturing cell and gene therapies, worth less than $20 billion in 2024, to expand rapidly as approvals drive higher volumes of production.
Breakout Ventures’ focus on early-stage companies stands out as more and more investors elect to save their dollars for derisked assets.
Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according to analysts at Stifel.
Single-trial approvals are raising the bar on trial design and execution. The new paradigm is pushing sponsors to plan earlier, step up their data and risk‑based quality management and use modeling and AI to generate one compelling, regulator‑ready evidence package.
AbbVie’s foray into the obesity space is successful so far, analysts agree, as amylin analog ABBV-295 elicited as much as 9.73% weight loss at 13 weeks in the multiple ascending dose portion of a Phase 1 trial.
FEATURED STORIES
Stylus Medicine, a member of BioSpace’s NextGen Class of 2026, launched in May 2025 to develop new, less complex genetic medicines. The company’s in vivo approach has attracted “intense” interest from Big Pharma.
Industry groups have identified upfront costs as a barrier to streamlining U.S. drugmaking. The nonprofit API Innovation Center has a proposed answer for how to tilt finances in favor of investments in continuous manufacturing.
A “significant number of patients” could see a cure for multiple myeloma within the next two decades, one expert told BioSpace. Here are five therapies that could change the treatment paradigm.
TrumpRX and DTC sales may expand prescription drug access, but they will not solve the affordability crisis by themselves.
Psychedelics are a “game changer” in depression care, according to William Blair, but the complicated treatment regimens mean they will likely be supplanted by more-traditional options once they become available.
UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for the therapy, perhaps even based on precedent from the recent drama surrounding Moderna’s mRNA flu vaccine.
LATEST PODCASTS
In our anniversary episode, we discuss a rare earnings miss for Eli Lilly, a pivotal metabolic dysfunction–associated steatohepatitis victory for Novo Nordisk’s Wegovy, growing excitement about CAR Ts for autoimmune disease and the ongoing controversy over HeLa cells.
Pfizer, Sanofi and others report Q3 beats; AbbVie, Roche and Novartis strike big deals; the 2024 presidential election looms; and BioSpace takes a look back at 10 years of NextGen, our annual pick of young biotechs to watch.
In this episode of Denatured, Lori and guests from Teva Pharmaceuticals and TOWER Capital review the investment landscape this year and the policies influencing investment heading into 2025.
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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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Servier will pick up Ojemda, which received FDA approval in 2024 to treat pediatric glioma. The drug clocked sales of $155 million for Day One Biopharmaceuticals in 2025. -
The alliance, which pairs Tenaya’s modality agnostic target identification and validation capabilities with Alnylam’s deep experience in RNA interference therapeutics, comes during a period of resurgence for the cardiovascular space. -
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. -
Sanofi will gain global exclusive rights over rovadicitinib, an oral JAK/ROCK blocker that has anti-inflammatory and anti-fibrotic effects. -
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.
WEIGHT LOSS
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The companies have been embroiled in a row about compounded GLP-1 drugs that escalated to a lawsuit last month. The legal action has now been dropped and the former adversaries have struck a deal that could increase access to Novo’s obesity medicines. -
Hansoh’s olatorepatide achieved 19% weight loss at 48 weeks in a Phase 3 trial in China, handing partner Regeneron a glimmer of hope for a pipeline in desperate need of reinvigoration. -
Zealand Pharma’s shares fell 32% in early morning trading Friday after its Roche-partnered asset petrelintide failed to meet investor expectations in a mid-stage clinical trial. -
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. -
Merck’s Keytruda will soon lose exclusivity, just as weight-loss giants Eli Lilly and Novo Nordisk press in with their blockbuster GLP-1s.
POLICY
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The settlement, which requires Moderna to pay the plaintiffs $950 million upfront plus up to $1.3 billion in contingent commitments, is an outcome “better than feared,” according to analysts. -
Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies. -
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. -
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. -
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.
In a competitive job market, how applicants present themselves in interviews is critical. Asking about promotions and expressing dislike for the work they’d be doing are just a few reasons hiring managers don’t extend job offers.
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.
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
IN CASE YOU MISSED IT
Center for Biologics Evaluation and Research Director Vinay Prasad will exit the FDA for the second time after a controversial run that saw the oncologist butt heads with colleagues and ruffle feathers in biopharma after presiding over the rejection of several rare disease therapies.
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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Roche’s shares tumbled nearly 5% on news that a key pillar in the five-pronged clinical plan for the breast cancer asset giredestrant was unsuccessful. -
Ipsen will withdraw Tazverik’s follicular lymphoma and epithelioid sarcoma indications as emerging data point to an elevated safety risk in patients undergoing treatment. -
The FDA’s cancer advisors will discuss AstraZeneca’s application for the oral SERD camizestrant in breast cancer and the AKT inhibitor Truqap in prostate cancer. -
Incyte is looking to expand the use of its PD-1 blocker Zynyz to treat non-small cell lung cancer. The FDA’s rejection did not flag problems with the drug’s efficacy or safety. -
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.
NEUROSCIENCE
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Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy. -
The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took issue with were filed in mid-2024. -
In this episode of Denatured, you’ll listen to Ram May-Ron, managing partner at FreeMind Group, and Ravi Kiron, managing director at Biopharma Strategy Advisors. We’ll be speaking about how to combine nondilutive funding and family office money into a unified strategy that gets companies through the drug development valley of death. -
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. -
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.
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. -
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. -
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.
