With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
Sana Biotechnology is looking to start clinical development for its type 1 diabetes therapy SC451 this year.
Follow along as BioSpace tracks job cuts and restructuring initiatives.
Dozens of biotechs reported earnings this week. BioSpace recaps key highlights from Capricor Therapeutics, Legend Biotech, Inovio and Allogene.
Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.
Small-molecule drugs account for nearly half of the most valuable investigational therapies for orphan diseases, according to analytics firm Evaluate.
Total assets under management for Novo Holdings, Novo Nordisk’s controlling shareholder, fell by more than one-third last year. The report caps off a tumultuous year for the Novo group of companies.
FEATURED STORIES
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.
While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
As the Formula 1 season kicks off in the APAC region, biopharma companies can take a page from the popular sport’s playbook around precision, adaptability, and the right team. In return, biopharma can also offer some lessons to elite motor sport on well-rounded excellence in the race to market.
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question whether shared evidence and prior knowledge will accelerate development in rare diseases.
The U.S. Senate has a plan to improve drug development for rare disease patients. The exit of controversial CBER chief Vinay Prasad will help clear the path.
Whether happening in public or private, biopharma M&A is fiercer than ever. Experts point to patent pressures, herd mentality and a declining stock of available biotechs with mature assets.
LATEST PODCASTS
In this episode of Denatured, you’ll listen to Oxana Iliach, senior director of regulatory strategy at Certara and Vera Pomerantseva, director of product management for risk-based quality management at eClinical Solutions. We speak about how the FDA’s latest decision to have one, rather than two pivotal studies, for new drug applications raises the bar for data collection and risk-based management.
Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.
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.
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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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Combining tirzepatide with vitamin B12, a common additive in compounded versions of the drug, yields an impurity that could alter the drug’s toxicity profile and pose safety risks to patients, the company said in an open letter. -
Orforglipron, Eli Lilly’s oral obesity drug, is under FDA review with a decision expected in April. The pharma has also filed for marketing authorization for the pill in China. -
Among the unreported adverse events potentially linked to Ozempic are two deaths and one case of “completed suicide,” according to an FDA inspection report. -
Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial. -
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.
POLICY
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The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.” -
TrumpRX and DTC sales may expand prescription drug access, but they will not solve the affordability crisis by themselves. -
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.
While you should never rely solely on AI tools when applying for jobs, they can greatly benefit the application process. Recruiting expert Bryan Blair discusses how using large language models can set you apart from the competition and includes a prompt framework to get you started.
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.
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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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. -
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.
NEUROSCIENCE
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UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp. -
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 LoomsWhile 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. -
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.
CELL AND GENE THERAPY
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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. -
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. -
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 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.
