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The struggles of gene therapies continue. Here is a very nice article where Biospace interviews Courtney Rice regarding the commercial status of marketed gene therapies.

Heather MacKenzie of BioSpace writes:

” Last year, just 64 patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT)—for which Casgevy was greenlit in January 2024—received infusions of the gene therapy, according to Vertex’s full-year 2025 earnings report. An additional 147 people had their first cell collection. Meanwhile, Genetix Bio—formerly bluebird bio—has treated over 100 patients with Lyfgenia, which is only approved for SCD, a member of Genetix’s executive team told BioSpace in an email.”

Foreseeing low adoption rates was somehow overlooked when modeling the economics early on as gene therapies were heralded as scientific breakthroughs.

What wasn’t taken into consideration was the patient journey required for conditioning prior to treatment, a process that is lengthy, painful and could lead to infertility.

“You’re asking a lot of time for these patients to be out of the game, both from the conditioning time to the procedure to the engraftment,” Rice said of the new gene therapies. “And that somehow sort of got glossed over in the parade, in the champagne shaking and excitement.”

I recall Stuart Orkin once say – there is a difference between treating a disease and relieving a disease burden. The two shouldn’t get mixed up.

Will adoption increase in the future? Time will tell, but many pharmas loath carrying low-profit drugs that generate inefficient returns on the bottom line.

The full article is worth reading here –

https://lnkd.in/eTC58tMN

Big Pharma, 12 months, $9 billion deal volume.
All on in vivo CAR therapy.Last week I covered Lilly’s acquisition of Orna therapeutics. Zoom out 12 months and you’ll see Big Pharma isn’t betting on one delivery platform. They’re buying all of them one after the other.

March 2025 to February 2026 tells the story:

• AstraZeneca grabbed EsoBiotec ($1B)
• AbbVie took Capstan Tx ($2B)
• Kite acquired Interius ($0.35B) then signed a partnership with Pregene for $1.64B
• BMS signed for Orbital at $1.5B
• Lilly closed ORNA last week ($2.4B)

What this pattern reveals? No one knows which delivery tech will dominate yet. But given these deals volume there must be promising data Big Pharma has seen at closed doors.

LVV. AAV. LNP. Hybrid platforms. They’re all in play.

If the winning platform was obvious, we’d see consolidation around one modality.

Instead, we’re seeing diversification.

Despite fundamental delivery challenges I’ve written about before remain unsolved:
• High cell-specific targeting.
• Controlled CAR expression.
• Safety at scale.

Data reveal that in vivo CAR-T might be a promising modality to fight B-cell driven hematological malignancies or autoimmune disease.

For RNA-LNP strategies there’s certainly room:
You don’t need durable CAR persistence to reset dysregulated B cells and you benefit a cleaner safety profile with transient expression.

Anyways, right now the race is wide open.

And this is why the next 24 months matter so much. Clinical data will separate the platforms that work from the ones that looked good on paper.

And the companies that solve delivery with a clean safety profile will command valuations that make $9 billion look conservative.

The question isn’t whether in vivo CAR-T will work. Big Pharma already answered that with their checkbooks.

The question is which platform gets there first.
Whoever does, will have a significant edge on this new wave of cell therapy.

What’s your read? Are we looking at the future winner in this chart, or is it in someone’s lab or still a draft on a blackboard?

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I share insights on all things xRNA, CAR-X and CGT innovation. Follow me for more biotech insights