Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the year, M&A is back with one deal for a gene editing biotech reinvigorating that sector; and RFK Jr. installs a suite of new vaccine board members who share his skeptical views on vaccines.
The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay.
Nearly $30 million in tax incentive awards are expected to add 1,519 new life sciences jobs in Massachusetts, with about 52% of the positions spread over Boston and Cambridge. Vertex, which received the largest award, will create hundreds of roles in Boston.
Jefferies analysts said on Tuesday that Ventyx could leverage its mid-stage data for VTX3232 to position the oral drug candidate as a treatment for other neurodegenerative diseases, including Alzheimer’s.
The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets.
Venclexta, when combined with azacitidine, elicited an overall survival benefit below 10% in patients with myelodysplastic syndromes.
FEATURED STORIES
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
As companies roll out data showing the power and improved safety profile of antibodies that target two antigens, analysts say the class could overtake monoclonal antibody Keytruda as the “immunotherapy backbone” of solid tumor treatment.
Large pharmaceutical companies were out in force at this week’s 2024 Cell & Gene Meeting on the Mesa, as they look to expand their presence in the industry.
Eli Lilly CEO David Ricks is confident his company and peer Novo Nordisk are years ahead in the weight loss space, as biotechs press on with compelling data.
Big Pharma can’t seem to get enough radiopharmaceutical biotechs. With Lilly, Sanofi and BMS chasing Novartis into the complex space, all eyes are on these specialty biotechs.
Tim Hunt, CEO of the Alliance for Regenerative Medicine, said Monday at the 2024 Cell & Gene Meeting on the Mesa that investments reached $10.9 billion in the first half of this year—outpacing 2019’s $9.8 billion total—but far below the pandemic peak.
FROM BIOSPACE INSIGHTS
Establishing trust through thought leadership is no longer optional in today’s cautious biopharma market. Learn how strategic insights and targeted outreach can turn awareness into high-converting leads.
LATEST PODCASTS
This week, we discuss the two major FDA approvals for sickle cell from Vertex/CRISPR and bluebird bio; Axcella and the future of long-covid treatments, Vanda’s $100m purchase and AI regulatory developments in Europe.
In this episode we dive into regulation, real-time management, and AI’s various applications and how it can streamline different processes with guests from Microsoft and IQVIA.
In this episode, hear from senior leaders at Microsoft and IQVIA to get their take on how generative AI is impacting productivity, employee engagement and how to mitigate risks.
Job Trends
Recently, GenScript Biotech Corporation, a world leader in technologies and services for life science R&D and manufacture, released its 2023 Environmental, Social, and Governance report.
Subscribe to Genepool
Subscribe to BioSpace’s flagship publication including top headlines, special editions and life sciences’ most important breaking news
SPECIAL EDITIONS
BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the second quarter of 2025, with increased pressure from further layoffs.
BioSpace did a deep dive into executive pay, examining the highest compensation packages, pay ratios and golden parachutes—what a CEO would get paid to leave.
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
DEALS
-
The neuroscience space saw a late push in merger and acquisition activity, while oncology start-ups reeled in the most venture capital funds. Industry leaders expect these trends to continue. -
Analysts say Novo Holdings made the right decision last week in scooping up the CDMO to increase manufacturing capacity for Novo Nordisk’s diabetes and weight-loss drugs. -
Gilead Sciences announced Monday it will purchase CymaBay Therapeutics and its investigational treatment seladelpar for primary biliary cholangitis, a type of liver disease that causes bile-duct damage. -
Shares of preclinical genetic medicines company Metagenomi tanked more than 30% on Friday afternoon in a disappointing debut for its initial public offering, bucking the trend of positive IPOs so far this year. -
Despite not having a single candidate in the clinical stage, the Moderna-backed biotech is offering 6.25 million shares for $15 apiece in an initial public offering. Shares are expected to begin trading Friday.
WEIGHT LOSS
-
Pfizer selects its candidate for the oral GLP-1 race as Eli Lilly strives to overtake Novo Nordisk in the injectable weight-loss drug space. Meanwhile, pressure builds to reduce drug prices in the U.S. -
To help keep pace with the demand for GLP-1 therapies, CordenPharma has announced a sizeable $980 million investment in its U.S. and European sites. -
Some analysts say so, and a recent study suggested Lilly’s tirzepatide beat Novo’s semaglutide at inducing weight loss, but there are other factors in the market race. -
Despite recent concerns about suicidality and other neuropsychiatric issues, a recent study has found that Novo Nordisk’s Ozempic (semaglutide) is associated with lower risks of dementia, cognitive deficit and nicotine misuse. -
Pfizer said Thursday it is pushing ahead with a once-daily, modified-release formulation of its oral GLP-1 obesity therapy danuglipron, with dose optimization studies in the second half of 2024.
POLICY
-
BridgeBio’s Attruby wins approval for transthyretin amyloid cardiomyopathy while the FDA accepts Alnylam’s application for Amvuttra in the indication; Cassava’s controversial Alzheimer’s drug flunks Phase III; Amgen’s MariTide fails to impress investors, Donald Trump’s controversial nominations continue.
-
Currently, Eli Lilly’s GLP-1 drugs Mounjaro and Zepbound (tirzepatide) are not on the FDA’s shortage list but compounded pharmacies are still making them. That’s unprecedented. -
Career conservative and former congressman Dave Weldon will, if confirmed, act as director of the Centers of Disease Control and Prevention, where his anti-vaccine views will mesh with those of selected Department of Health and Human Services head Robert F. Kennedy Jr. -
RFK Jr. as HHS head is perhaps President-elect Donald Trump’s most controversial Cabinet pick now that Matt Gaetz has withdrawn as nominee for Attorney General. With Dr. Oz tapped to lead CMS and maybe Marty Makary at the FDA, it’s going to be quite the show. -
Makary is a pancreatic surgeon at Johns Hopkins who became known for battling medical mistakes and in recent years has been an outspoken critic of COVID-19 policies.
A career in regulatory affairs combines knowledge of law, science, economics and more. Those who follow this biotech career path aren’t necessarily scientists, but they do need to understand the science behind the products that they need to regulate.
Are you currently working full time and in school earning a master’s degree? If so, do learn these time management skills to balance your personal and professional life.
Establishing boundaries between your personal and professional lives is the key to maintaining fulfilling versions of each.
Is your job is slowly starting to make you feel unmotivated or unproductive? Are you unable to work properly? Use these motivation tips to rejuvenate yourself.
As we leave the winter of 2020-21 behind, let’s learn more about the strategies to help college graduates secure employment during COVID-19.
Let’s review the six tips for finding work during the coronavirus pandemic.
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
-
With the regulatory approval for advanced breast cancer, Roche’s inavolisib is a potential challenger to Novartis’ PI3K inhibitor Piqray, which last year generated $505 million in revenue. -
The overall survival data from the late-stage trial will help Pfizer in its bid to expand the label for Talzenna and Xtandi, potentially covering all patients with metastatic castration-resistant prostate cancer regardless of biomarker status. -
The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia. -
After several high-profile failures, including BMS’ $1.5B breakup with Agenus, anti-TIGIT therapies are generating cautious optimism. -
Opdivo’s approval for patients with resectable non-small cell lung cancer comes as the regulator recently raised concerns of overtreatment with this type of therapeutic regimen with platinum-doublet chemotherapy.
NEUROSCIENCE
-
Patient assistance programs may actually be a two-way street, providing patients with drugs and companies with data. -
Two CRLs from the FDA last week cited concerns with third-party manufacturers, while Indian CDMOs may make a bid for U.S. business if there is a decoupling from Chinese companies under the BIOSECURE Act. -
Donanemab, which will be marketed as Kisunla, will compete with Biogen and Eisai’s Leqembi. -
After back-to-back failures in 2021, Wave Life Sciences has finally aced a Phase Ib/IIa Huntington’s disease trial and is looking to a potential accelerated approval for its investigational antisense oligonucleotide. -
The FDA’s calendar is relatively light in July, with only five major deadlines, including one for a PD-1 blocker and another for an opioid overdose drug.
CELL AND GENE THERAPY
-
BioSpace will be in attendance at the American Society of Gene & Cell Therapy’s 27th annual meeting, along with thousands of others. Stay with us for updates throughout the week. -
Cell and gene therapy company Cellectis announced Monday it has completed a $140 million investment from AstraZeneca that extends the biotech’s cash runway into 2026. -
Jeffrey Chamberlain, president of the American Society of Gene & Cell Therapy, spoke with BioSpace about what we can expect to learn about in Baltimore this week.
-
Adeno-associated viruses have long been go-to vectors for gene therapies. How AAVs are improving will be among the cell and gene therapy topics to be covered in Baltimore this week. -
Follow News Editor Greg Slabodkin and Managing Editor Jef Akst as they travel with some 8,000 others for discussions of cell and gene therapy advances, challenges, regulations and more.
