Gene/Cell Therapy Quarterly Update: June 2023

Critical updates in an ever-changing environment

June 22, 2023

This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialty drugs, biosimilar drugs and  traditional drugs. 

New Drug Information

  • Elevidys® (delandistrogene moxeparvovec-rokl): The U.S. Food and Drug Administration (FDA) granted accelerated approval of Sarepta Therapeutics’ Elevidys, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMDElevidys is a one time treatment that addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells. The Phase 1 ENDEAVOR trial, demonstrated an improvement of 3.8 points from baseline on the North Star Ambulatory Assessment (NSAA) at one year, compared to external controls, following a single infusion of Elevidys. The Phase 3 EMBARK study of 120 boys with DMD ages 4 through 7 years, which will assess change from baseline in the NSAA total score after one year compared to placebo, is expected to readout in the latter half of 2023.1 Sarepta set the price at $3.2 million per one time Elvidys administration.

New Indications

  • None



FDA approved Gene/Cell Therapy Products
Gene and Cell Therapy Pipeline (Submitted to the FDA)
Generic Name Brand Name Manufacturer Indication(s) Route of Administration PDUFA date Prevalence in the US
SRP9001 (delandistrogene moxeparvovec) N/A Sarepta Therapeutics/


Ambulatory duchenne muscular dystrophy (DMD) IV June 2023 ~4,500
valoctocogene roxaparvovec Roctavian™ BioMarin Severe hemophilia A IV June 2023 8/100,000 males
remestemcel-L N/A Mesoblast Children with steroid-refractory acute graft-versus-host disease IV August 2023 TBD


NurOwn BrainStorm Cell Therapeutics Amyotrophic lateral sclerosis


Intrathecal September 2023 ~30,000
lifileucel N/A Iovance Unresectable or metastatic melanoma CAR-T November 2023 ~180,000
exagamglogene autotemcel (CTX-001) N/A Vertex Sickle cell disease (SCD) IV December 2023 20,000
TDT April 2024 1,500
lovotibeglogene autotemcel N/A bluebird bio SCD IV December 2023 20,000
FDA approved Gene/Cell Therapy Products
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved  
delandistrogene moxeparvovec-rokl Elevidys® Sarepta Therapeutics/


Ambulatory duchenne muscular dystrophy (DMD) IV June 2023  
beremagene geperpavec-svdt Vyjuvek® Krystal Biotech Dystrophic epidermolysis bullosa (DEB) Topical May 2023  
omidubicel-onlv Omisirge® Gamida Cell Blood cancers in need of allogeneic hematopoietic stem cell transplant (HSCT) Cell therapy April 2023  
nadofaragene firadenovec-vncg Adstiladrin® Ferring and FKD Therapies Oy Certain forms of bladder cancer Catheter into bladder December 2022  
etranacogene dezaparvovec-drlb Hemgenix™ CSL Behring Moderate to severe hemophilia B IV November 2022  
elivaldogene autotemcel Skysona™ bluebird Bio Cerebral adrenoleukodystrophy (CALD) IV September 2022    
betibeglogene autotemcel Zynteglo® bluebird Bio Transfusion-dependent beta-thalassemia (TDT) IV August 2022    
ciltacabtagene autoleucel Carvykti™ Johnson & Johnson (Janssen) R/R multiple myeloma CAR-T February 2022  
allogeneic processed thymus tissue adgc Rethymic™ Enzyvant Congenital athymia Surgical October 2021  
idecabtagene vicleucel Abecma™ Bristol-Myers Squibb and bluebird bio R/R multiple myeloma CAR-T March 2021  
lisocabtagene maraleucel Breyanzi™ Bristol-Myers Squibb R/R large B cell lymphoma (LBCL) IV February 2021  
brexucabtagene autoleucel Tecartus™ Kite R/R Mantle cell lymphoma R/R acute lymphoblastic leukemia (ALL) CAR-T July 2020  
onasemnogene abeparvovec-xioi Zolgensma AveXis Spinal muscular atrophy IV May 2019  
voretigene neparvovec-rzyl Luxturna® Spark Therapeutics Biallelic RPE65 mutation-associated retinal dystrophy Subretinal injection December 2017  
axicabtagene ciloleucel Yescarta® Kite R/R LBCL/follicular lymphoma (FL) IV October 2017  
tisagenlecleucel Kymriah® Novartis R/R ALL/LBCL IV August 2017  


Extended Gene Therapy* 2023-2025 Pipeline
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Filing date* Prevalence
atidarsagene autotemcel


Libmeldy™ Orchard Metachromatic leukodystrophy (MLD) IV 2023 ~460
eladocagene exuparvovec Upstaza™ PTC Therapeutics Aromatic L-amino acid decarboxylase (AADC) deficiency Intracerebral 3Q2023 300 in world
AGTC-501 (laruparetigene zosaparvovec) N/A AGTC X-linked retinitis pigmentosa Subretinal 2H2023 20,000 in US and Europe
botaretigene sparoparvovec N/A J & J and


X-linked retinitis pigmentosa Subretinal 1H2024 20,000 in US and Europe
fidanacogene elaparvovec N/A Roche/Pfizer Moderate to severe hemophilia B IV 2H2023 1.5/100,000 males
lenadogene nolparvovec


Lumevoq™ GenSight Biologics Leber hereditary optic neuropathy Intravitreal injection 2H2023 6.5/million
OTL-201 N/A Orchard MPS IIIA IV 2H2023 ~1,000
LYS-SAF-302 (olenasulfligene relduparvovec) N/A Lysogene MPS IIIA Intracerebral 2H2023 ~1,000

(prademagene zamikeracel)

N/A Abeona Recessive DEB Topical/surgical – One time treatment 2H2023 400
fordadistrogene movaparvovec N/A Pfizer DMD IV 4Q2023 9,000-12,000
RP-L201 (marnetegragene autotemcel) N/A Rocket Severe leukocyte adhesion deficiency type 1 (LAD-1) IV 4Q2023 25-50/year severe
Fanconi Anemia 6.25/million

(rebisuligene etiparvovec)

N/A Ultragenyx MPS IIIA IV 1Q2024 ~1,000
UX-701 N/A Ultragenyx Wilson disease IV 1H2024 ~9,000

(giroctocogene fitelparvovec)

N/A Sangamo/


Severe hemophilia A IV 3Q2024 8/100,000 males

(verbrinacogene setparvovec)

N/A Freeline Therapeutics Moderate to severe hemophilia B IV 2H2024 1.5/100,000 males
GS030 N/A GenSight Retinitis pigmentosa Intravitreal 2H2024 20,000 in US and Europe
pariglasgene brecaparvovec N/A Ultragenyx Glycogen storage disease type 1a IV 2H2024 8/million


N/A Regenxbio Wet age-related macular degeneration Subretinal 1H2025 1.1 million

(bidridstrogene xeboparvovec)

N/A Sarepta Limb girdle muscular dystrophy type 2E IV 1H2025 3.5/million
RGX-121 N/A Regenxbio Hunter’s syndrome Intracisternal 1H2025 <1/million
AMT-130 N/A uniQure Huntington’s disease Intracerebral 2H2025 15.2/million


*Gene Therapy: One-time administration with curative intent and anticipated WAC of > $750,000

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