Big Three Saturday: Robust Funding & Partnerships, HHS Chaos Continues, and RNA + Gene-Editing Updates

Editor’s Note Preamble:

When it is ever not a busy news week? Over Christmas?

For today’s trend analysis, what we’re discussing from the past week:

1) Biotech funding still going on - with some big rounds to cover
2) RNA & gene-editing therapies making the rounds
3) US Regulatory Chaos continues

Let me know what you think of these Saturday trends write-ups via email or on LinkedIn.

Robust Funding Activity Despite the Uncertainty

Despite all the headwinds and regulatory uncertainty, biotech funding remained strong across multiple sectors and geographies.

Several companies secured substantial late-stage financing, including Mineralys Therapeutics' $250 million public offering for its hypertension program and Galvanize Therapeutics' oversubscribed $100 million Series C for its cancer and lung disease treatments.

International markets showed particular strength. European biotechs attracted significant investment, with London-based CHARM Therapeutics raising $80 million for its AI-designed menin inhibitor and Belgium's MRM Health securing €55 million for microbiome-based therapeutics. Swedish biotech Modus Therapeutics completed an oversubscribed unit issue worth approximately SEK 28.3 million for their polysaccharide program across anemia and other inflammatory conditions.

There were also a few notable deals with substantial upfront payments. Novartis committed $200 million upfront to Arrowhead Pharmaceuticals for its preclinical siRNA Parkinson's disease program, with the total deal potentially worth $2 billion. Servier committed $210 million upfront to IDEAYA Biosciences for their small molecule protein kinase C inhibitor in a rare eye cancer, while Vertex paid $45 million upfront to Enlaza Therapeutics in a deal potentially worth over $2 billion, focusing on drug conjugates & T cell engagers towards autoimmune disease.

This continued diversity in funding and partnerships suggests continued investor and institutional confident in biotech fundamentals, despite political and regulatory volatility.

Notable RNA and Gene Editing Updates

Therapeutics around and involving RNA were in the spotlight this week, with a few notable deals and clinical read-outs being announced. One stand-out is Novartis’ $160 million upfront deal with China-based Argo Biopharma for cardiovascular RNA medicines, as well as Novartis’ previously mentioned partnership with Arrowhead Pharmaceuticals for its siRNA Parkinson's program.

Clinical data provided mixed but encouraging signals for the RNA editing field. Wave Life Sciences reported results from its alpha-1 antitrypsin deficiency program, with its RNA editing therapy WVE-006 achieving regulatory thresholds for approval despite some investor concerns about the magnitude of effects. The company's approach represents the first RNA editing therapy to reach human testing.

Gene editing also returned to the headlines, with Editas Medicine nominating EDIT-401 as its lead in vivo development candidate as part of a massive strategic pivot. The company reported EDIT-401 to have achieved 90% mean reduction in LDL cholesterol in nonhuman primates and plans to submit IND filings by mid-2026.

US Healthcare Political Upheaval Continues

As is the unfortunate norm these day, politic discourse continued to dominate pharma and healthcare headlines, reaching an unprecedented fever pitch.

Last’s weeks firing of CDC Director Susan Monarez by HHS RFK Jr., purportedly due to her refusal to support rescinding certain COVID-19 vaccine approvals, has resulted in a cascade of resignations from senior CDC officials. These include Chief Medical Officer Deb Houry and the directors of the National Center for Emerging and Zoonotic Infectious Diseases and the National Center for Immunization and Respiratory Diseases.

The FDA has simultaneously implemented significant policy changes under CBER Director Vinay Prasad, who announced requirements for studies "roughly 5 to 10 times larger than historically accepted" for future vaccine approvals. Prasad also mandated randomized controlled trials for vaccine co-administration claims, departing from previous reliance on immunogenicity data.

The series of upheavals has drawn criticism from multiple quarters. Over 1,000 current and former HHS employees signed a letter calling for Kennedy's resignation, while 21 major medical organizations, including the Infectious Diseases Society of America and the American Public Health Association, issued a joint statement demanding his departure.

And during a contentious Senate Finance Committee hearing, lawmakers from both parties questioned Kennedy's leadership, with some suggesting he poses a danger to public health.

That’s all for this weekly trend overview - now go enjoy your weekend (despite all the chaos going on).