Cell and Gene Therapy Today—April 23, 2026

This week’s Gene and Cell Therapy update highlights regulatory momentum, pivotal clinical progress, strategic acquisitions, manufacturing expansion, and platform partnerships across neurology, oncology, and rare disease.

In Today’s Newsletter

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🧬 Roche starts new Elevidys Phase 3 after EMA setback [1] [EU • 16 Apr 2026]

https://www.fiercepharma.com/pharma/roche-launch-another-elevidys-study-after-eu-rejection-duchenne-gene-therapy
Context: Elevidys (Roche, Sarepta Therapeutics; Duchenne muscular dystrophy gene therapy) received a negative EMA opinion in ambulatory patients; Roche is now planning a new global Phase 3 trial.
Key point: Roche said the new placebo-controlled study in about 100 ambulatory boys is intended to generate additional data for a European resubmission.
Implication: May influence prescriber choice and payer reviews pending full data.

👁️ J&J returns bota-vec rights to MeiraGTx [2] [US • 16 Apr 2026]

https://www.fiercebiotech.com/biotech/jj-pockets-25m-refund-meiragtx-struggling-eye-disease-gene-therapy
Context: Botaretigene sparoparvovec (MeiraGTx; X-linked retinitis pigmentosa gene therapy) previously failed a Phase 3 study on vision-guided mobility.
Key point: Johnson & Johnson transferred rights back to MeiraGTx for $25 million upfront, while MeiraGTx said it plans to pursue U.S. and EU filings.
Implication: May influence prescriber choice and payer reviews pending full data.

🏭 Andelyn Biosciences and ENCell build a US–APAC manufacturing bridge [3] [US • 21 Apr 2026]

https://www.prnewswire.com/news-releases/andelyn-biosciences-and-encell-co-ltd-announce-strategic-partnership-to-expand-global-reach-of-gene-therapy-manufacturing-302745085.html
Context: Andelyn Biosciences (US CDMO) and ENCell (South Korea CDMO) announced a strategic manufacturing collaboration for gene therapy programs.
Key point: The companies said the agreement creates a US–APAC production corridor to support local manufacturing for regional clinical trials and broader global expansion.
Implication: May expand screening, initiation, and follow-up at scale.

❤️ Medera wins FDA Fast Track for AAV-SERCA2a in DMD cardiomyopathy [4] [US • 16 Apr 2026]

https://www.manilatimes.net/2026/04/16/tmt-newswire/globenewswire/medera-receives-fda-fast-track-designation-for-gene-therapy-targeting-duchenne-muscular-dystrophy-associated-cardiomyopathy/2321940
Context: AAV-SERCA2a (Medera; gene therapy for Duchenne muscular dystrophy-associated cardiomyopathy) is being evaluated in the first-in-human MUSIC-DMD trial.
Key point: Medera said the FDA granted Fast Track Designation for AAV-SERCA2a, enabling closer regulatory interaction and potential expedited review pathways.
Implication: Introduces a regulatory catalyst that may shape development planning and review timelines.

🫁 Genprex expands biomarker work for REQORSA [5] [US • 21 Apr 2026]

https://www.morningstar.com/news/pr-newswire/20260421da39074/genprex-signs-sponsored-research-agreement-to-study-biomarkers-that-may-predict-patient-response-to-reqorsa-gene-therapy
Context: REQORSA (Genprex; quaratusugene ozeplasmid) is in development for non-small cell lung cancer and small cell lung cancer, including the Acclaim-1 and Acclaim-3 studies.
Key point: Genprex signed a sponsored research agreement with MD Anderson to study TROP2 and PTEN as potential biomarkers of response.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧠 UCB buys Neurona to enter regenerative neurology [6] [EU • 17 Apr 2026]

https://www.fiercebiotech.com/biotech/ucbs-115b-deal-neurona-touches-several-ambitions-including-strategic-expansion-advanced
Context: Neurona Therapeutics’ lead asset, NRTX-1001, is a neuronal cell therapy in Phase 1/2 testing for drug-resistant mesial temporal lobe epilepsy.
Key point: UCB agreed to acquire Neurona for $650 million upfront plus up to $500 million in milestones, citing expansion into regenerative medicine and advanced therapies.
Implication: Signals pipeline investment and modality expansion.

🚶 Kyverna reports registrational miv-cel win in stiff person syndrome [7] [US • 21 Apr 2026]

https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-presents-registrational-trial-primary-analysis-miv-cel
Context: Miv-cel (Kyverna Therapeutics; autologous CAR-T) was studied in the single-arm registrational Phase 2 KYSA-8 trial in stiff person syndrome.
Key point: Kyverna reported statistically significant improvement across primary and secondary endpoints at 16 weeks, with all treated patients off chronic immunotherapies at Week 16.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 ImmunoScape shows preclinical Seed-and-Boost data at AACR [8] [21 Apr 2026]

https://www.biospace.com/press-releases/immunoscape-presents-data-on-significant-selective-in-vivo-expansion-of-cell-therapy-product-at-aacr-annual-meeting
Context: ImmunoScape presented preclinical data for its WT1-targeted Seed-and-Boost platform, which combines tumor-specific T cells with a boosting molecule for in vivo expansion.
Key point: The company reported selective T-cell expansion, tumor infiltration, and survival benefits in animal models, with a first-in-human investigator-initiated trial planned for Autumn 2026.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🔄 Circio and Acuitas team up on circular RNA in vivo CAR-T [9] [16 Apr 2026]

https://www.drugtargetreview.com/circio-and-acuitas-partner-on-circular-rna-car-t-therapy/2135220.article
Context: Circio Holding ASA is contributing its circVec circular RNA platform, while Acuitas Therapeutics is contributing lipid nanoparticle delivery technology.
Key point: The companies will evaluate circular RNA-based in vivo CAR-T approaches for cancer and autoimmune disease, aiming for longer expression than conventional mRNA approaches.
Implication: Signals pipeline investment and modality expansion.

💰 Lilly buys Kelonia to deepen in vivo CAR-T push [10] [US • 20 Apr 2026]

https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-kelonia-therapeutics-advance-vivo-car-t-cell
Context: Kelonia Therapeutics’ lead program, KLN-1010, is a Phase 1 in vivo BCMA CAR-T gene therapy for relapsed/refractory multiple myeloma.
Key point: Lilly agreed to acquire Kelonia for up to $7.0 billion, including $3.25 billion upfront, to add the iGPS in vivo gene placement platform and expand genetic medicine capabilities.
Implication: Signals pipeline investment and modality expansion.

Why It Matters

Duchenne muscular dystrophy remains a major cell and gene therapy battleground, with Roche restarting evidence generation for Elevidys in Europe and Medera advancing a cardiomyopathy-focused program [1][4].
In vivo cell therapy is moving from concept to capital deployment, highlighted by Lilly’s Kelonia deal and Circio–Acuitas platform work [9][10].
Neurology is becoming a larger advanced-therapy market, with UCB buying Neurona in epilepsy and Kyverna posting registrational data in stiff person syndrome [6][7].
Manufacturing and supply-chain strategy is becoming a competitive lever, as shown by the Andelyn–ENCell US–APAC collaboration [3].
Precision approaches are still expanding around gene therapy, including biomarker enrichment work for Genprex’s REQORSA program [5].

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FAQ

What changed for Elevidys in Europe?

Roche said it will run a new placebo-controlled Phase 3 trial in ambulatory Duchenne muscular dystrophy patients after the EMA previously issued a negative opinion. The new study is intended to support a resubmission in Europe [1].

Why did Johnson & Johnson return bota-vec to MeiraGTx?

Bota-vec previously missed a key Phase 3 endpoint in X-linked retinitis pigmentosa. J&J returned the asset, while MeiraGTx said it still plans to seek U.S. and EU approvals (regulatory outcome not stated) [2].

What is notable about Kyverna’s miv-cel data in stiff person syndrome?

Kyverna said the registrational KYSA-8 trial met all primary and secondary endpoints, with durable mobility and disability improvements and discontinuation of chronic immunotherapies by Week 16. The company is preparing a BLA submission [7].

What did Lilly gain by acquiring Kelonia Therapeutics?

Lilly gains Kelonia’s iGPS platform for generating CAR-T cells inside the body and its lead program KLN-1010 in Phase 1 for relapsed/refractory multiple myeloma. The deal also broadens Lilly’s in vivo genetic medicine footprint [10].

What is UCB getting from Neurona Therapeutics?

UCB is adding NRTX-1001, a neuronal cell therapy in Phase 1/2 for drug-resistant mesial temporal lobe epilepsy. UCB framed the deal as both an epilepsy expansion and a move into regenerative medicine [6].

Why does the Andelyn–ENCell partnership matter?

The companies said the agreement links US and APAC manufacturing to support local production for clinical trials and future commercial supply. That could reduce logistical friction for cross-region gene therapy programs [3].

Entities / Keywords

Elevidys (delandistrogene moxeparvovec, Duchenne muscular dystrophy gene therapy)
Roche, Sarepta Therapeutics, EMA, Duchenne muscular dystrophy, ambulatory DMD
Botaretigene sparoparvovec (bota-vec), MeiraGTx, Johnson & Johnson, X-linked retinitis pigmentosa
Andelyn Biosciences, ENCell, CDMO, gene therapy manufacturing, US–APAC
Medera, Sardocor, AAV-SERCA2a, MUSIC-DMD, DMD-associated cardiomyopathy
Genprex, REQORSA (quaratusugene ozeplasmid), MD Anderson, TROP2, PTEN, Acclaim-1, Acclaim-3
UCB, Neurona Therapeutics, NRTX-1001, mesial temporal lobe epilepsy, regenerative medicine
Kyverna Therapeutics, miv-cel (mivocabtagene autoleucel, KYV-101), KYSA-8, stiff person syndrome
ImmunoScape, Seed-and-Boost, WT1, TCR-T, AACR 2026
Circio, circVec, Acuitas Therapeutics, circular RNA, in vivo CAR-T
Eli Lilly, Kelonia Therapeutics, KLN-1010, iGPS, BCMA, multiple myeloma