Drug Pricing Returns, AI Labs Scale Up, Gene Therapy Pushes Mainstream – This Week in Biotech #85
Trump’s healthcare framework, the NVIDIA–Lilly discovery engine, IPO green shoots, and the latest regulatory and clinical signal checks (Jan 9-15, 2026).
Welcome back to This Week in Biotech by Biotech Blueprint, edition 85, covering biotech and pharma news from January 9th to 15th, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Washington is back in the drug pricing conversation. President Trump’s “Great Healthcare Plan” wants to pull some U.S. drug prices closer to what other wealthy countries pay through “most favored nation” pricing, squeeze middlemen economics by targeting pharmacy benefit manager kickbacks, and push transparency so patients can actually see prices and coverage in plain English. The part to watch is execution as lower prices only translate into better access if savings show up at the pharmacy counter and in out of pocket costs.
NVIDIA and Eli Lilly are building an AI co-innovation lab and planning up to one billion dollars over five years for talent, compute, and infrastructure. The pitch is a closed loop engine that links automated wet labs with modeling so experiments generate data, data improves models, and models choose the next experiments.
Eikon’s initial public offering filing is an early sign that the market may be warming to new biotech listings again, with the real test coming at pricing and trading. In parallel, Beam laid out a cleaner 2026 plan with a longer runway into 2029 and a stated path toward a biologics license application filing for its sickle cell program as early as the end of 2026. The common thread is that the market is rewarding credible timelines again, but only when the milestones are concrete and close enough to touch.
And none of it matters until regulators say it does. Atara took a second Complete Response Letter for Ebvallo, with the agency now saying the single arm dataset is no longer sufficient to demonstrate efficacy, even as manufacturing issues were described as resolved. On the flip side, ImmunityBio is pairing real commercial momentum with fresh lung cancer proof points: rising Anktiva revenue in bladder cancer, new Saudi approvals for lung cancer and bladder cancer, and updated lung cancer data that supports the company’s argument that Anktiva can help restore immune competence when added to checkpoint inhibitors.
Gene therapy put up more evidence that it can compete in large indications, not just ultra rare ones. Ocugen posted an interim look in geographic atrophy with lesion growth reduction and clean early safety, while Krystal showed delivery and expression signals in the lung for cystic fibrosis. Regenxbio added longer follow up in Duchenne and kept its accelerated approval timeline in view. The message is breadth as delivery is improving and the modality is creeping into mainstream markets.
BIOTECH/PHARMA NEWS 🧬
🔹 Trump unveiled the “Great Healthcare Plan,” a broad framework that asks Congress to do three things: lock in “most favored nation” drug pricing deals meant to bring some United States prescription prices closer to what other wealthy countries pay, change exchange subsidies by sending assistance directly to eligible people rather than insurers while also funding the program that lowers out of pocket costs for some lower income enrollees, and force more transparency and less middleman economics by targeting pharmacy benefit manager kickbacks and requiring insurers and Medicare or Medicaid participating providers to post clearer pricing and coverage information.
President Trump’s Great Healthcare Plan is a framework urging Congress to lock in his Most Favored Nation drug pricing agreements, expand over-the-counter access for verified safe medicines, and redirect federal subsidy dollars away from insurers and directly to eligible Americans. It also proposes funding the Affordable Care Act cost-sharing reduction program, which the White House says would save taxpayers at least $36B and cut premiums for the most common Obamacare plans by over 10% per the Congressional Budget Office, while banning pharmacy benefit manager kickbacks that flow to brokerage middlemen. The plan would require insurers to present coverage and rates in plain English and disclose claim payouts versus profits, denial rates, and routine-care wait times, and it would require any provider or insurer that takes Medicare or Medicaid to post pricing and fees prominently.
🔹 NVIDIA and Eli Lilly are launching an AI co-innovation lab in the Bay Area and plan to invest up to $1B over five years in talent, compute, and infrastructure. The lab will use NVIDIA BioNeMo and next-gen NVIDIA architecture (Vera Rubin) to build a “closed-loop” discovery setup that links automated wet labs with computational modeling so experiments and models continuously improve each other. They also plan to push robotics and physical AI beyond discovery into manufacturing, using tools like digital twins to optimize production and supply chains.
🔹 ImmunityBio paired commercial momentum with more lung cancer proof points: the company said preliminary Q4 2025 net product revenue was $38.3M (up 20% vs. Q3 and 431% year over year), driven by continued Anktiva uptake in bladder cancer, with full-year 2025 preliminary net product revenue of $113M (the company says 700% year over year) and unit volume up 750% vs. 2024. It ended 2025 with $242.8M in cash and marketable securities and flagged recent Saudi approvals as incremental catalysts, including Anktiva plus a checkpoint inhibitor for metastatic non-small-cell lung cancer and Anktiva plus standard bladder therapy for high risk, treatment resistant non-muscle invasive bladder cancer. On the clinical side, updated lung cancer data suggested Anktiva may help rebuild immune competence when added to checkpoint inhibitors like Keytruda or Opdivo: in a first-line study, the combo increased absolute lymphocyte counts vs. checkpoint inhibitor alone, and in a later-line single-arm study, 77% of patients restored or maintained lymphocyte counts above a predefined threshold, with greater lymphocyte recovery tracking with longer survival.
🔹 Fortress jumped after the FDA approved Zycubo (copper histidinate, formerly CUTX-101), the first U.S.-approved copper replacement therapy for pediatric Menkes disease, a rare and often fatal disorder. Under Cyprium’s deal with Sentynl (Zydus), Cyprium will receive tiered royalties on net sales plus up to $129M in milestones, and it will also receive the Rare Pediatric Disease Priority Review Voucher issued with the approval.
🔹 Atara sank after the FDA issued a second Complete Response Letter for Ebvallo (tabelecleucel) in EBV-positive post-transplant lymphoproliferative disease. This time, the FDA said the single arm ALLELE study is no longer adequate to show efficacy and that design, conduct, and analysis issues limit interpretability, even though the agency confirmed the prior GMP deficiency is now resolved and raised no new safety concerns. Atara and partner Pierre Fabre said they will seek a Type A meeting to clarify a path forward, but the decision reintroduces major regulatory uncertainty for a product already approved in Europe (as Ebvallo).
🔹 Beam Therapeutics rallied after outlining a cleaner 2026 plan and longer cash runway. It said it ended 2025 with about $1.25B in cash and expects funding into 2029, supporting a biologics license application filing for risto-cel in sickle cell disease as early as year end 2026. Beam also said it aligned with the FDA on a potential accelerated approval path for BEAM-302 in alpha-1 antitrypsin deficiency using biomarker endpoints, with updated phase 1/2 data and a pivotal plan due by the end of the first quarter of 2026, plus more 2026 data for BEAM-301 and a new liver program reveal in the first half of 2026.
🔹 Moderna’s stock has pushed back above $40 per share, a level it only touched briefly in January 2025 and otherwise has not held since late 2024. The move came as the company reiterated a 2026 plan for up to 10% revenue growth by building a “seasonal vaccine” business, while continuing aggressive cost cuts and positioning its cash engine to fund the next wave of oncology and rare disease programs. Management also pointed to about $1.9B in expected 2025 revenue, a lower 2025 operating expense outlook, and an estimated about $8.1B year end cash balance, with a stated goal of reaching cash breakeven in 2028, plus multiple 2026 clinical and regulatory catalysts. CEO Stéphane Bancel framed it as stabilization plus discipline: fewer expenses, a clearer commercial lane, and enough runway to keep taking shots on the pipeline.
🔹 Eikon Therapeutics filed on January 9 to go public on Nasdaq under EIKN, a decent read through on whether biotech’s rebound is strong enough to reopen the IPO window. The Merck-vet-led company (Roger Perlmutter, with Roy Baynes as CMO) is selling investors on a late stage oncology pipeline led by EIK1001, a systemic TLR7/8 agonist in a global phase 2/3 melanoma trial with pembrolizumab and additional NSCLC studies, plus PARP1 inhibitors EIK1003/1004 in phase 1/2 across multiple solid tumors, with proceeds aimed at advancing these programs through key 2026 milestones.
CLINICAL TRIAL UPDATES 📊
🔹 Ocugen said its one-time gene therapy OCU410 for geographic atrophy (dry AMD) cut lesion growth by 46% at 12 months in an interim phase 2 look (about half of patients evaluated so far), with no treatment-related serious adverse events reported across phase 1/2. The company plans to share full phase 2 data later this year and start phase 3 in 2026.
🔹 Regenxbio shares rose after it reported new 18 month phase 1/2 data for its Duchenne gene therapy RGX-202. In four patients treated at the pivotal dose, all exceeded the expected disease trajectory on the North Star Ambulatory Assessment, improving an average of 7.4 points versus the established progression model (and 6.6 points at 12 months). Regenxbio said it expects pivotal topline results in early Q2 2026 and plans to file a BLA under the accelerated approval pathway in mid 2026.
🔹 Vir Biotechnology shared an update in chronic hepatitis delta: updated phase two SOLSTICE data showed its combination of tobevibart and elebsiran was well tolerated and drove hepatitis delta virus ribonucleic acid to “target not detected” in 88% of evaluable participants at 96 weeks, versus lower rates on antibody alone.
🔹 Krystal Biotech shared a positive interim update from its phase 1 CORAL-1 study of KB407, an inhaled gene therapy for cystic fibrosis designed to deliver normal, full length CFTR protein to the lungs. In the highest dose cohort, lung biopsies confirmed delivery and expression in all six patients with successful bronchoscopies, with about 29% to 42% of conducting airway cells showing transduction signals. Safety looked consistent with earlier cohorts, with mostly mild to moderate, transient adverse events and one serious asthma event judged related to the bronchoscopy procedure rather than the drug. Krystal has submitted the design for a registrational repeat-dosing study, CORAL-3, to the FDA and expects to start enrollment in the first half of 2026 after alignment.
Have a great rest of your week and thanks for reading Biotech Blueprint!
Great roundup of the week's developments. The Beam update caught my attention because extending the runway to 2029 while maintaining a near-term BLA path for risto-cel shows they're balancing fiscal discipline with clinical momentum. What's particularly interesting is the biomarker-based accelerated approval pathway they secured for BEAM-302 in AATD, that kind of regulatory alignment can be really valuable when you're tryingto compress development timelines. I saw similar dynamics play out with other genetic medicine companies where cash management becamse the key differentiator between those who made it to approval and those who didn't.