FDA approvals - March 2026
New options emerged in PBC-associated itch, plaque psoriasis, neurologic Hunter syndrome, and platinum-resistant ovarian cancer.
FDA approves Rocket's Kresladi as the first gene therapy for severe leukocyte adhesion deficiency-I
The first gene therapy for severe LAD-I achieves 100% HSCT-free survival with sustained CD18 expression through 42 months in paediatric patients lacking a matched donor
Brad Sitko, CIO, XOMA Royalty | Onyx Live | JPM 2026
The biotech industry destroys billions in shareholder value every year - failed trials, depleted cash, and abandoned pipelines leaving investors with nothing.
Avi Veidman, CEO, Nucleai | Onyx Live | JPM 2026
90% of oncology drugs fail in clinical trials - costing billions and, more importantly, delaying life-saving treatments for patients who need them now.
Will biotech still be relevant in 2030? | Treehill Partners Ep. 3 | JPM 2026
Will large pharma's AI adoption and deployment to drug development redefine biotech's role and business model?
Paul Denslow, CEO, Intus Bio | Onyx Live | JPM 2026
38 trillion bacteria live in your gut — yet most microbiome tests can only identify them at the genus level, missing the strains that actually matter.
Mike Cordonnier, CEO, Carlsmed | Onyx Live | JPM 2026
Hundreds of thousands of spine fusions are performed each year, but implant choice is still often limited to standard sizes.
Chris Hall, CEO, Personalis | Onyx Live | JPM 2026
Personalis is betting that detecting relapse at the molecular level, before it becomes radiographically obvious, can change how cancer is monitored and managed.
Slone Partners’ Leslie Loveless on the post-boom talent reset in biopharma
Biopharma’s boom-era org charts are giving way to leaner, faster operating models. Slone Partners CEO, Leslie Loveless, says the edge now goes to pragmatic builders: “The companies that win right now are hiring leaders who can do more with less.”
A world-expert in Stargardt disease has developed a pill to slow vision loss: why Tinlarebant could win FDA approval
After years studying Stargardt’s natural history, Belite Bio’s Dr. Hendrik Scholl is championing Tinlarebant as a practical early intervention - “Intervention is simply one pill a day - and we are able to slow down the progression of disease by more than one-third.”
Belite Bio concludes phase 3 Stargardt trials with nearly 40% retinal cell preservation
Belite is positioning Tinlarebant for regulatory submission after Phase 3 Stargardt data, emphasizing the long-term safety burden of a systemic, lifelong oral therapy: “For a lifelong treatment, the safety bar is about as high as it gets.”
FDA approvals - January 2026
Copper replacement formalizes care for a fatal pediatric metabolic disorder.
“Efficacy with safety”: the north star behind Outpace’s next-gen engineered cell therapies
Outpace Bio is using AI-powered protein design to build “software and hardware” into engineered cells aimed at durable solid-tumor responses. For CEO Marc Lajoie, “efficacy is still the fundamental problem that needs to be solved - efficacy and safety.”
Why Novellia thinks the best biopharma data strategy starts with patients, not pipelines
“Everything that’s good for patients delivers extraordinary value for biopharma - that overlap is the entire company,” says Shashi Shankar, CEO. From symptom tracking to AI-driven normalization, Novellia builds patient-first data infrastructure that scales into biopharma-grade intelligence.
The lab-data marketplace powering precision medicine’s next wave
As pharma’s demand for real-time lab intelligence accelerates, Prognos Health is leaning in - and after six or seven years of building, the company now sees “a real path to delivering 20 billion health insights by 2050”, says CEO Sundeep Bhan.