Gene

Therapy
Report Q1 2024-Q4 2026
Projected Treatments and Launch Timelines
 2024 PROJECTED LAUNCHES The 2024 pipeline includes treatments for multiple myeloma and leukemia.

QUARTER THERAPY MANUFACTURER PHASE OF TYPE BREAKTHROUGH DRUG INDICATION ROUTE OF ESTIMATED
NAME DEVELOPMENT THERAPY CLASS ADMINISTRATION POTENTIAL U.S.
DESIGNATION & FREQUENCY CANDIDATES

1Q Breyanzi Bristol-Myers Pending FDA Supplemental No Chimeric The treatment of relapsed or Injection-IV, 145,000
(lisocabtagene Squibb approval indication antigen refractory chronic lymphocytic one-time additional
receptor
maraleucel) 03/14/2024 leukemia or small lymphocytic adult patients
(CAR) T-cell
lymphoma in adults who have
therapy,
received a prior Bruton tyrosine
ex vivo
kinase inhibitor and B-cell
lymphoma 2 inhibitor

1Q Abecma Bluebird Bio/ Pending FDA Supplemental No CAR T-cell The treatment of adults with Injection-IV, 57,000
(idecabtagene Bristol-Myers approval indication therapy, relapsed or refractory multiple one-time additional
vicleucel) Squibb/Celgene 03/16/2024 ex vivo myeloma who have received 2 to adult patients
3 prior lines of therapy

1Q atidarsagene Orchard Pending FDA New biologic No Gene The treatment of metachromatic Injection-IV, 100–600
autotemcel Therapeutics approval therapy, leukodystrophy in patients ages one-time pediatric
03/18/2024 ex vivo 6 years and younger with late patients
infantile form without clinical
manifestations, and in patients
ages 6 years and younger with
early juvenile form without clinical
manifestations or with early clinical
manifestations of the disease

1Q Kresladi Rocket Pending FDA New biologic No Gene The treatment of severe leukocyte Injection-IV, 150 pediatric
(marnetegragene Pharmaceuticals approval therapy, adhesion deficiency type 1 in one-time patients
autotemcel, 03/31/2024 ex vivo patients ages 3 months and older
fka RPL201)

2Q Carvykti Janssen Pending FDA Supplemental No CAR T-cell The treatment of relapsed or Injection-IV, 130,000
(ciltacabtagene Pharmaceuticals/ approval indication therapy, refractory multiple myeloma in one-time additional
autoleucel) Johnson & 04/05/2024 ex vivo patients who have received 1 to 3 adult patients
Johnson prior lines of therapy

2Q fidanacogene Pfizer/Spark Pending FDA New biologic Yes Gene The treatment of severe Injection-IV, 1,300 adult
elaparvovec Therapeutics approval therapy, hemophilia B in adults one-time patients
04/27/2024 in vivo

Gene Therapy Report Q1 2024–Q4 2026 Source: RxPipeline, CVS Health Clinical Affairs. Information current as of February 21, 2024. 2
 2024 CONTINUED More than a dozen new therapies could gain approval in 2024.

QUARTER THERAPY MANUFACTURER PHASE OF TYPE BREAKTHROUGH DRUG INDICATION ROUTE OF ESTIMATED
NAME DEVELOPMENT THERAPY CLASS ADMINISTRATION POTENTIAL U.S.
DESIGNATION & FREQUENCY CANDIDATES

2Q prademagene Abeona Pending FDA New biologic Yes Gene The treatment of recessive Surgical graft, 450 adult
zamikeracel Therapeutics approval therapy, dystrophic epidermolysis bullosa one-time and pediatric
05/25/2024 ex vivo in patients ages 6 years and older patients

2Q Elevidys Sarepta Pending FDA Supplemental No Gene The treatment of ambulatory Injection-IV, 370 additional
(delandistrogene Therapeutics approval indication therapy, patients ages 6 to 7 years with one-time pediatric
moxeparvovec- 06/22/2024 in vivo Duchenne muscular dystrophy males
rokl) with a confirmed mutation in the
DMD gene

4Q obecabtagene Autolus Pending FDA New biologic No CAR T-cell The treatment of relapsed Injection-IV, 21,000 adult
autoleucel Therapeutics approval therapy, or refractory B-cell acute one-time patients
11/16/2024 ex vivo lymphoblastic leukemia in adults

4Q dabocemagene Castle Creek Phase III New biologic No Gene The treatment of recessive Injection- 450 adult
autoficel Pharma therapy, dystrophic epidermolysis bullosa Intradermal, and pediatric
ex vivo in patients ages 7 years and older multi-dose patients

4Q RPL102 Rocket Phase II New biologic No Gene The treatment of Fanconi anemia Injection-IV, <1,000
Pharmaceuticals therapy, in patients ages 1–17 years one-time pediatric
ex vivo patients

4Q UX111 Abeona Phase III New biologic No Gene The treatment of Injection-IV, 1,500–4,000
Therapeutics/ therapy, mucopolysaccharidosis type one-time adult and
Ultragenyx in vivo IIIA (also known as Sanfilippo pediatric
Pharmaceutical syndrome type A) patients

4Q zevorcabtagene CARsgen Phase I/II New biologic No CAR T-cell The treatment of relapsed or Injection-IV, 45,000 adult
autoleucel Therapeutics therapy, refractory multiple myeloma after one-time patients
ex vivo at least 3 prior systemic therapies
in adults

Gene Therapy Report Q1 2024–Q4 2026 Source: RxPipeline, CVS Health Clinical Affairs. Information current as of February 21, 2024. 3
 2025 PROJECTED LAUNCHES New treatments for hemophilia A and cutaneous melanoma could be approved in 2025.

QUARTER THERAPY MANUFACTURER PHASE OF TYPE BREAKTHROUGH DRUG INDICATION ROUTE OF ESTIMATED
NAME DEVELOPMENT THERAPY CLASS ADMINISTRATION POTENTIAL U.S.
DESIGNATION & FREQUENCY CANDIDATES

1Q Breyanzi Bristol-Myers Phase II Supplemental No CAR T-cell The treatment of adults Injection-IV, 35,000–51,000
(lisocabtagene Squibb indication therapy, with relapsed or refractory one-time additional
maraleucel) ex vivo follicular lymphoma or marginal adult patients
zone lymphoma

1Q Upstaza PTC Phase II New biologic No Gene The treatment of aromatic Injection- <50 pediatric
(eladocagene Therapeutics therapy, L-amino acid decarboxylase Intracerebral, patients
exuparvovec) in vivo deficiency in patients ages one-time
17 years and younger

2Q fordadistrogene Pfizer Phase III New biologic No Gene The treatment of ambulatory Injection-IV, 850 pediatric
movaparvovec therapy, patients with Duchenne one-time males
in vivo muscular dystrophy

2Q pariglasgene Ultragenyx Phase III New biologic No Gene The treatment of glycogen Injection-IV, 3,000 adult
brecaparvovec Pharmaceutical therapy, storage disease type 1a in patients one-time and pediatric
in vivo ages 8 years and older patients

2Q resamirigene Astellas Pharma Phase I/II New biologic No Gene The treatment of X-linked Injection-IV, 40 male
bilparvovec therapy, myotubular myopathy in males one-time newborns
in vivo younger than 5 years per year

2H vusolimogene Replimune Phase I/II New biologic No Gene The treatment of cutaneous Injection- 13,000 adult
oderparepvec Group Inc. therapy, melanoma after progression on Intratumoral, patients
in vivo anti-PD1 therapy, in combination multi-dose
with Opdivo (nivolumab)

3Q giroctocogene Pfizer/Sangamo Phase III New biologic No Gene The treatment of hemophilia A Injection-IV, 3,000 adult
fitelparvovec BioSciences therapy, in adults one-time patients
in vivo

3Q RGX121 RegenxBio Phase III New biologic No Gene The treatment for Injection- < 25 pediatric
therapy, mucopolysaccharidosis type II, Intracerebral, patients
in vivo also known as Hunter syndrome, in one-time
patients ages 5 years and younger

4Q botaretigene Johnson & Phase III New biologic No Gene The treatment of X-linked Injection- 2,800–6,400
sparoparvovec Johnson/ therapy, retinitis pigmentosa due to RPGR Intraocular, adult and
MeiraGTx in vivo mutations in patients ages 3 years one-time pediatric
and older patients

Gene Therapy Report Q1 2024–Q4 2026 Source: RxPipeline, CVS Health Clinical Affairs. Information current as of February 21, 2024. 4
 2026 PROJECTED LAUNCHES A gene therapy to treat knee osteoarthritis, which affects millions, could get approval in 2026.

QUARTER THERAPY MANUFACTURER PHASE OF TYPE BREAKTHROUGH DRUG INDICATION ROUTE OF ESTIMATED
NAME DEVELOPMENT THERAPY CLASS ADMINISTRATION POTENTIAL U.S.
DESIGNATION & FREQUENCY CANDIDATES

1Q anitocabtagene Arcellx, Inc./ Phase II New biologic No CAR T-cell The treatment of relapsed or Injection-IV, 45,000 adult
autoleucel Gilead Sciences/ therapy, refractory multiple myeloma after one-time patients
(fka CARTddBCMA) Kite ex vivo at least 3 prior systemic therapies
in adults

1Q avalotcagene Ultragenyx Phase III New biologic No Gene The treatment of ornithine Injection-IV, 3,600–5,700
ontaparvovec Pharmaceutical therapy, transcarbamylase deficiency in one-time adult and
in vivo patients ages 12 years and older pediatric
patients

1Q Invossa Kolon Group Phase III New biologic No Gene The treatment of Injection- 15.9 million
(tonogenchoncel-L) therapy, knee osteoarthritis Intra-articular, adult patients
in vivo one-time

1Q ProstAtak Advantagene/ Phase III New biologic No Gene The first-line treatment of adults Injection- 73,800 adult
(aglatimagene Candel therapy, with intermediate to high risk, Intratumoral, patients
besadenovec) Therapeutics in vivo localized prostate cancer, in multi-dose
combination with external beam
radiation therapy and valacyclovir

1Q Zolgensma AveXis/Novartis Phase III New No Gene The treatment of spinal muscular Injection- 3,900
(onasemnogene formulation therapy, atrophy type 2 in patients ages Intrathecal, pediatric
abeparvovec-xioi) in vivo 2–17 years one-time patients

2Q VTX801 Pfizer/Vivet Phase I/II New biologic No Gene The treatment of hepatolenticular Injection-IV, 6,500–8,600
Therapeutics therapy, degeneration (Wilson’s Disease) one-time adult patients
in vivo in adults

2H OCU400 Ocugen Phase I/II New biologic No Gene The treatment of retinitis Injection- 3,600–5,700
therapy, pigmentosa associated with Intraocular, adult and
in vivo NR2E3 and RHO mutations and one-time pediatric
the treatment of Leber congenital patients
amaurosis associated with
CEP290 mutations in patients
ages 6 years and older

Gene Therapy Report Q1 2024–Q4 2026 Source: RxPipeline, CVS Health Clinical Affairs. Information current as of February 21, 2024. 5
 2026 CONTINUED The 2026 pipeline includes a gene therapy for wet age-related macular degeneration.

QUARTER THERAPY MANUFACTURER PHASE OF TYPE BREAKTHROUGH DRUG INDICATION ROUTE OF ESTIMATED
NAME DEVELOPMENT THERAPY CLASS ADMINISTRATION POTENTIAL U.S.
DESIGNATION & FREQUENCY CANDIDATES

4Q RGX314 AbbVie/ Phase III New biologic No Gene The treatment of neovascular (wet) Injection- 2 million adult
RegenxBio therapy, age-related macular degeneration Intraocular, patients
in vivo one-time

4Q RPA501 Rocket Phase II New biologic No Gene The treatment of Danon disease in Injection-IV, 7,500–15,000
Pharmaceuticals therapy, males ages 8 years and older one-time adult and
in vivo pediatric
patients

These breakthrough therapies offer great clinical potential, though at a high cost. Plan sponsors have a lot to
consider in terms of coverage, affordability, and long-term value.
Read our Insights post, “Gene and Genetically Modified Cellular Therapies: Coverage Considerations for Payors,”
to learn more.

Information compiled from external sources. Manufacturer drug launch dates are subject to change without notice. Some products may not be dispensed by CVS Specialty Pharmacy.
This document contains references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with CVS Health.
Source: RxPipeline, CVS Health Clinical Affairs. Information current as of February 21, 2024.
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