GENE

THERAPY
LANDSCAPE
APPROVED THERAPIES IN US
Luxturna
Developed By: Luxturna (voretigene neparvovec) works by delivering a functional copy of the RPE65 gene to overcome the genetic mutation and restore the vital RPE65 enzyme.
This allows the visual cycle to resume its normal function, leading to improved vision in patients with RPE65-associated retinal dystrophy. Luxturna’s is anticipated
to generate an estimated overall revenue of USD 326 million

Targeted Gene:
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Delivery Vector:
RPE65 2017 USD 850,000 each eye Retinal Dystrophy Modified AAV
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Zolgensma
Developed By: Zolgensma (onasemnogene abeparvovec) works by delivering a functional copy of the SMN1 gene to motor neurons, correcting the genetic defect that causes SMA.
This leads to increased SMN protein production, which helps maintain motor neuron health and improves motor function in patients with SMA. The drug generated
USD 1370 Million in 2022.

Targeted Gene:
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Delivery Vector:
SMN1 2019 USD 2.1 million Spinal Muscular Atrophy scAAV9

Zynteglo
Developed By: Zynteglo (Betibeglogene autotemce) works by correcting the underlying genetic defect in the β-globin gene in hematopoietic stem cells. This leads to increased
production of red blood cells containing functional β-globin protein, potentially eliminating the need for lifelong red blood cell transfusions for patients with TDT.

Targeted Gene:
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β-globin 2022 USD 2.8 Million Beta-Thalassemia BB305 LVV

2023 Gene Therapy Landscape 2

APPROVED THERAPIES IN US
Skysona
Developed By: Skysona (elivaldogene autotemcel) works by delivering a functional copy of the ABCD1 gene to cells in the brain and nervous system. This helps restore normal
myelin production and repair damaged nerve fibers, potentially slowing the progression of CALD and improving neurological function.

Targeted Gene:
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Approval Year:
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Treatment Cost:
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Indication: Delivery Vector:
ABCD1 2022 USD 3 Million Cerebral
. Lentiviral Vector
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Adrenoleukodystrophy

Hemgenix
Developed By: Hemgenix (etranacogene dezaparvovec) works by delivering a functional FIX gene to liver cells, enabling the production of FIX protein and restoring normal blood
clotting function. This leads to improved bleeding control and the potential for a cure for hemophilia B. Adstiladrin does not alter any existing genes, making it a
unique and targeted therapy.

Targeted Gene:
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Delivery Vector:
Factor IX 2022 USD 3.5 million Hemophilia B Modified AAV

Adstiladrin
Developed By: Adstiladrin (Nadofaragene firadenovec) works by delivering a gene encoding IFNα2b to the bladder urothelium. This helps to restore the immune response and
leads to anti-tumor effects, which can result in the regression of bladder tumors.

Targeted Gene:
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IFNα2b 2022 USD 63,190 Non-Muscle Invasive

.
Modified AAV
Bladder Cancer

2023 Gene Therapy Landscape 3

APPROVED THERAPIES IN US
Elevidys
Developed By: Elevidys (delandistrogene moxeparvovec) works by delivering a modified version of the dystrophin gene to muscle cells, leading to increased dystrophin production
and improved muscle function in patients with Duchenne muscular dystrophy.

Targeted Gene:
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Approval Year:
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Treatment Cost:
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Indication: Delivery Vector:
dystrophin 2023 USD 3.2 Million Duchenne muscular
. Modified AAV
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dystrophy

Vyjuvek
Developed By: Vyjuvek (beremagene geperpavec) works by delivering a functional copy of the COL7A1 gene to skin cells, leading to increased type VII collagen production,
improved skin integrity, and enhanced wound healing in patients with epidermolysis bullosa. Vyjuvek is first re-dosable gene therapy that is delivered through
topical application

Targeted Gene:
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COL7A1 2023 USD 630,500 annually Dystrophic Epidermolysis

. herpes-simplex virus type
Bullosa 1 vector

Roctavian
Developed By: Roctavian (valoctocogene roxaparvovec) works by delivering a functional FVIII gene to liver cells, enabling the production of FVIII protein and restoring normal
blood clotting function. This leads to improved bleeding control and the potential for a cure for hemophilia A.

Targeted Gene:
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Factor VIII 2023 USD 2.9 Million Hemophilia A AAV5

2023 Gene Therapy Landscape

UPCOMING THERAPIES IN US
Pre-Registration Phase
CASGEVY PF-06838435
Developed By: Indication: Developed By: Indication:
Generic Name:
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Exagamglogene autotemcel Sickle Cell disease, Fidanacogene Hemophilia B

Beta Thalessemia Elaparvovec
Expected Date for FDA Approval :
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Expected Date for FDA Approval :
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Q4 2023 - Q1 2024 Q4 2023 - Q1 2024

LentiGlobin KRESLADI
Developed By: Indication: Developed By: Indication:
Generic Name:
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Generic Name:
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lovotibeglogene autotemcel Sickle Cell disease marnetegragene autotemcel Severe LAD-I

Expected Date for FDA Approval :

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Q4 2023 - Q1 2024 Q4 2023 - Q1 2024

pz-cel (EB-101) Libmeldy

Developed By: Indication: Developed By: Indication:
Generic Name:
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Generic Name:
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prademagene Recessive Dystrophic atidarsagene Metachromatic

zamikeracel Epidermolysis Bullosa autotemcel leukodystrophy
Expected Date for FDA Approval :
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Q4 2023 - Q1 2024 Q1 2024 - Q2 2024

Abbreviation: RMAT - Regenerative Medicine Advanced Therapy, LAD-1- Leukocyte adhesion deficiency type 1
UPCOMING THERAPIES IN US
Pre-Registration Phase
FCX-007 PTC-AADC
Developed By: Indication: Developed By: Indication:
Designation:
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Fast Track Dystrophic Epidermolysis None aromatic L-amino acid

Bullosa decarboxylase deficiency
Expected Date for FDA Approval :
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Expected Date for FDA Approval :
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Q4 2023 - Q1 2024 Q1 2024 - Q2 2024

CG0070 PF-06939926
Developed By: Indication: Developed By: Indication:
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Designation:
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RMAT Non Muscular Invasive Orphan Drug, Fast Track, Duchenne Muscular
Bladder Cancer RMAT Dystrophy
Expected Date for FDA Approval :
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Q4 2024 - Q1 2025 Q1 2025 - Q2 2025

Engensis Generx
Developed By: Developed By: Indication:
Designation: Indication: Designation:
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None Diabetic Peripheral Orphan Drug Refractory Angina

Neuropathy
Expected Date for FDA Approval : Expected Date for FDA Approval :
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Q1 2025 - Q2 2025 Q2 2025 - Q3 2025

Abbreviation: RMAT - Regenerative Medicine Advanced Therapy, LAD-1- Leukocyte adhesion deficiency type 1
UPCOMING THERAPIES IN US
Late Clinical Phase
NFS-01 TG-C
Developed By: Indication: Developed By:
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Indication:
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None Leber Hereditary Optic Fast Track Degenerative Osteoarthritis

Neuropathy
Expected Date for FDA Approval :
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Q2 2025 - Q3 2025 Q2 2025 - Q3 2025

PF-06939926 AGTC 501

Developed By: Indication: Developed By: Indication:
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Orphan Drug, Breakthrough Hemophilia A Orphan Drug X-Linked Retinitis

Therapy Pigmentosa
Expected Date for FDA Approval :
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Q2 2025 - Q3 2025 Q2 2025 - Q3 2025

RGX-314 OTL-103
Developed By: Developed By: Indication:
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Indication:
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NA Wet Age-Related Macular RMAT Wiskott Aldrich Syndrome

Degeneration
Expected Date for FDA Approval :
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Expected Date for FDA Approval :
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Q2 2026 - Q3 2026 Q2 2026 - Q3 2026

Abbreviation: RMAT - Regenerative Medicine Advanced Therapy

UPCOMING THERAPIES IN US
Late Clinical Phase
GS010 SPK-8011
Developed By: Indication: Developed By:
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Indication:
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NA Leber Hereditary Optic Fast Track Hemophilia A

Neuropathy
Expected Date for FDA Approval :
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Expected Date for FDA Approval :
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Q3 2026 - Q4 2026 Q3 2027 - Q4 2027

Abbreviation: RMAT - Regenerative Medicine Advanced Therapy

UPCOMING TECHNOLOGIES
CRISPR–Cas9 gene editing therapy
Developed By: Casgevy represents an innovative gene therapy utilizing CRISPR/Cas9 gene editing to enhance the synthesis of fetal hemoglobin (HbF) within red blood
cells. HbF, a variant of hemoglobin, remains non-polymerized in low oxygen environments, offering potential relief from the symptoms associated with
transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). The Medicines and Healthcare products Regulatory Agency (MHRA)
has granted Casgevy a conditional marketing authorization. This milestone paves the way for anticipated approvals from the US Food and Drug
Administration on December 8 for sickle cell disease and on March 30, 2024, for β-thalassemia.

Zinc Fingers-based therapies

Developed By: Zinc finger proteins are small proteins that bind to specific sequences of DNA. They are named after their zinc finger domain, which is a structure that binds
to zinc ions. Zinc finger proteins can be engineered to bind to any sequence of DNA, making them a versatile tool for gene therapy. Zinc finger-based
therapies are still in the early stages of development, but they have the potential to treat a wide range of diseases. Sangamo Therapeutics is currently working
on multiple neurology indication using their proprietary library of thousands of zinc fingers. These Therapies are in early phases of development

megaTAL therapy
Developed By: 2seventy bio, is currently use the MegaTAL gene editing platform to develop bbT369. bbT369 targets a novel combination of antigens highly expressed in B
cell lymphomas, CD79a and CD20. CD79a, a novel target, is a critical signaling component of the B cell receptor and CD20 is a known clinical target for
lymphoma. The dual targeting of bbT369 may limit antigen escape as a mechanism of lymphoma relapse. and use cells gene-edited with megaTAL™
technology to remove the function of CBLB, a known negative regulator of T cells. Removal of CBLB function may enable robust antigen-dependent CAR
T cell expansion and allow cells to resist anergy and maintain activity in sub-optimal conditions for T cell activation.