Gene/Cell Therapy Quarterly Update: September 2023

Critical updates in an ever-changing environment

September 25, 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. 

FDA approved Gene/Cell Therapy Products in the last 12 months
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Month Approved  
valoctocogene roxaparvovec-rvox Roctavian™ BioMarin Severe hemophilia A IV June 2023  
delandistrogene moxeparvovec-rokl Elevidys® Sarepta Therapeutics/


Duchenne muscular dystrophy (DMD)

(4-5 years of age)

IV June 2023  
donislecel-jujn Lantidra® CellTrans Inc Uncontrolled type 1 diabetes Hepatic portal vein 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  
Gene and Cell Therapy Pipeline (Submitted to the FDA)
Generic Name Brand Name Manufacturer Indication(s) Route of Administration PDUFA Date Estimated Prevalence in the US


NurOwn® BrainStorm Cell Therapeutics Amyotrophic lateral sclerosis


Intrathecal December 2023 25,000
exagamglogene autotemcel N/A Vertex Sickle cell disease (SCD) IV December 2023 20,000
Transfusion dependent

beta-thalassemia (TDT)

April 2024 1,500
lovotibeglogene autotemcel N/A bluebird bio SCD IV December 2023 20,000
lifileucel N/A Iovance Unresectable or metastatic melanoma CAR-T cell therapy February 2024 180,000
atidarsagene autotemcel N/A Orchard Metachromatic leukodystrophy IV March 2024 2,500
fidanacogene elaparvovec N/A Roche & Pfizer Hemophilia B IV April 2024 3.9-6.5/million
Extended Gene Therapy* 2023-2026 Pipeline
Generic Name Brand Name Manufacturer Indication(s) Route of Administration Anticipated Filing date+ Estimated Prevalence in the U.S.
mametegragene autotemcel N/A Rocket Severe leukocyte adhesion deficiency type 1 (LAD-1) IV Submitted 25-50/year
eladocagene exuparvovec Upstaza™ PTC Therapeutics Aromatic L-amino acid decarboxylase (AADC) deficiency Intracerebral 3Q2023 300 in world

(prademagene zamikeracel)

N/A Abeona Recessive DEB Topical/surgical – One time treatment 2H2023 400
mozafancogene autotemcel N/A Rocket Fanconi Anemia IV 4Q2023 6.25/million
RGX-121 N/A Regenxbio Hunter’s syndrome Intracisternal 1H2024 <1/million
UX-701 N/A Ultragenyx Wilson disease IV 2H2024 9,000

(giroctocogene fitelparvovec)

N/A Sangamo/


Severe hemophilia A IV 3Q2024 8/100,000 males
fordadistrogene movaparvovec N/A Pfizer DMD IV 4Q2024 9,000-12,000
botaretigene sparoparvovec N/A J & J and


X-linked retinitis pigmentosa Subretinal 2H2024 20,000 in US and Europe

(verbrinacogene setparvovec)

N/A Freeline Therapeutics Moderate to severe hemophilia B IV 2H2024 1.5/100,000 males
pariglasgene brecaparvovec N/A Ultragenyx Glycogen storage disease type 1a IV 2H2024 8/million
lenadogene nolparvovec


Lumevoq™ GenSight Biologics Leber Hereditary Optic Neuropathy Intravitreal injection 1H2025 6.5/million

(bidridstrogene xeboparvovec)

N/A Sarepta Limb girdle muscular dystrophy type 2E IV 1H2025 3.5/million
AMT-130 N/A uniQure Huntington’s disease Intracerebral 2H2025 15.2/million
AGTC-501 (laruparetigene zosaparvovec) N/A AGTC X-linked retinitis pigmentosa Subretinal 2H2025 20,000 in US and Europe
OTL-201 N/A Orchard MPS IIIA IV 2026 1,000
PBGM-01 N/A Passage Bio GM1 gangliosidosis Intrathecal 2026 TBD
MYR101 N/A Myrtelle Canavan disease Intracerebro-ventricular 2026 TBD
RP-A501 N/A Rocket Danon Disease IV 2026 TBD


N/A Regenxbio Wet age-related macular degeneration Subretinal 2026 1.1 million

IV=intravenous; CAR=chimeric antigen receptor; TBD=to be determined

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

+Estimates based on clinical evidence available at the time of publication

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