Rating: Buy Sangamo Therapeutics, Inc. (SGMO) 2021 ...

Company UpdateHealthcare

May 19, 2021

Sangamo Therapeutics, Inc. (SGMO)Rating: Buy

Patrick R. Trucchio, CFA212-856-5749

[email protected]

STAAR of Pack on Track for Initial In-Human Data Late in2021; Establishing Fabry Disease Market Model; Reiterate Buy

Stock Data 05/18/2021Price $10.82Exchange NASDAQPrice Target $25.0052-Week High $19.4352-Week Low $8.51Enterprise Value (M) $966Market Cap (M) $1,557Public Market Float (M) 116.9Shares Outstanding (M) 143.93 Month Avg Volume 1,311,588Short Interest (M) 15.18Balance Sheet MetricsCash (M) $629.5Total Debt (M) $38.5Total Cash/Share $4.38Book Value/Share $6.13

Cash (M): Includes cash, cash equivalents, and investments.

EPS ($) DilutedFull Year - Dec 2019A 2020A 2021E1Q (0.41) (0.37) (0.32)A2Q (0.26) (0.26) (0.29)3Q (0.24) (0.01) (0.31)4Q 0.04 (0.29) (0.32)FY (0.85) (0.90) (1.25)Revenue ($M)Full Year - Dec 2019A 2020A 2021E1Q 8.1 13.1 26.3A2Q 17.5 21.3 32.53Q 22.0 57.8 32.54Q 54.9 25.8 32.5FY 102.4 118.2 123.8

2021 Annual: Note that quarterly revenues in 2021 primarily reflectmilestone and other payments that we have allocated equally perquarter.

20

18

16

14

12

10

8MAY-20 SEP-20 JAN-21 MAY-21

20

15

10

5

0

Vol. (mil) Price

ST-920 appears well-positioned in Fabry disease, in our view.Today, we have established market model assumptions in Fabrydisease in which we estimate gene therapies (GTs) could generateglobal peak annual revenues in excess of $4.5B or more than $30B inaggregate from 2H24 to 2035. We believe ST-920, Sangamo's wholly-owned GT and among the most advanced AAV-based treatments indevelopment, could vie for market leadership. We reiterate our $25 pricetarget and Buy rating on SGMO shares, which we find very compellingat current levels.

Initial STAAR trial data expected by end of 2021. The STAAR studyrepresents the first in human treatment with ST-920, a recombinantAAV2/6 vector encoding the cDNA for human α-Gal A (αGLA). Asa reminder, in preclinical studies, ST-920 showed supraphysiologicalαGLA activity in several key tissues including the kidney, heart,spleen, and liver of mice, and reduced lyso-Gb3 below 10% of thatseen in untreated mice within two months post-treatment. In STAAR,Sangamo is to evaluate safety and tolerability of ascending doses ofST-920, which aims to provide stable, long-term production of αGLAat therapeutic levels in subjects with Fabry disease. The constantproduction of αGLA in humans should, importantly, enable reductionand potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a singledose of ST-920 and followed for a period of 52 weeks, per the protocol.Separately, we note that on May 3, AVROBIO, a biotechnology companydeveloping AVR-RD-01, an ex vivo lentiviral gene therapy for Fabrydisease, provided an adverse program update; specifically, AVROBIOcan no longer pursue an accelerated approval with a Phase 2 trial (FAB-GT) and instead intends to discuss with FDA a registration trial witha primary efficacy endpoint of clearance of GL-3 (also referred to asGb3) inclusions in biopsied renal peritubular capillaries (PTCs) as thebasis for full approval. AVROBIO noted that on March 11, approximatelythree weeks before a planned End-of-Phase 1 meeting, FDA grantedfull approval of Fabrazyme (agalsidase beta), an enzyme replacementtherapy (ERT), more than 18 years after it received accelerated approvalon the basis of a surrogate endpoint: reduction of GL-3/Gb3 inclusionsin biopsied renal PTCs. Sangamo has noted the following: (1) threepatients are enrolled and dosed in STAAR, with a fourth patient enrolledwhom should be dosed soon; (2) Sangamo is collecting biomarker dataas well as conducting kidney biopsies at the appropriate time; (3) theAVROBIO update is not germane to the STAAR program; Sangamois collecting the necessary data for a potential full approval of ST-920in Fabry disease, including Gb3 reduction in the kidney and long-termeGFR benefit; (4) Sangamo is collecting data in a range of patientsincluding those on ERT, those naive to ERT, and those pseudo-naiveto ERT, which should provide a broad dataset with which to discuss thepath forward for the program with the FDA, which we believe could occurin 2022 after data from STAAR is generated.

Model update. We fine-tuned our ST-920 estimates to better reflect ournew market model assumptions. Refer to pages 18-20 of this report foradditional details.

For definitions and the distribution of analyst ratings, analyst certifications, and other disclosures, please refer to pages 25 - 28 of this report.

Valuation and risks. We have assessed Sangamo using a discounted cash flow (DCF)-based and sum-of-the-parts (SOTP)-based valuation methodology. We assign probabilities of success to SB-525 in hemophilia A of 60%, to ST-920 in Fabry diseaseof 45%, to ST-400 in beta thalassemia of 30%, and to SAR445136 in sickle cell disease of 25%. We model a build in continuedmilestone payments tied to Sangamo's ZFP platform from 2021 and onward, and revenues tied to Sangamo's ZFP platform inthe late 2020s, leading to a moderate present value. We employ a 12% discount rate and a terminal multiple of 6.0x. Takentogether, these assumptions yield a total firm value of approximately $3.7B and price target of $25. Risks include, but arenot limited to: (1) clinical development risk tied to SB-525 and ST-920, among other programs; (2) commercialization risk; (3)reimbursement risk; (4) capital markets and dilution risk; (5) pricing risk; and (6) risk from COVID-19 should a renewed wavelead to a re-enactment of broad lock-downs globally.

Sangamo Therapeutics, Inc. May 19, 2021

H.C. WAINWRIGHT & CO. EQUITY RESEARCH 2

Company Pipeline

Source: Company filings, H.C. Wainwright & Co.

$ in millions, unless noted

*Manufactured by ex vivo gene editing of a patient's own (autologous) hematopoietic stem cells using non-viral delivery

of zinc finger nuclease technology.

Program Indication Target Status Rights Prior New Prior New Year

ST-920 Fabry Disease Liver Phase 1/2 Wholly-owned 428 580 951 1,290 2030E

ST-101 PKU Liver Preclinical Wholly-owned

ST-400* β-Thalassemia Phase 1/2 448 448 1,492 1,492 2030E

SAR445136* Sickle Cell Disease (SCD) Phase 1/2 1,116 1,116 4,465 4,465 2031E

KITE-037 Oncology T cells Preclinical

TX200 Solid Organ Transplant Kidney Preclinical Wholly-owned

ST-501 Tauopathies CNS Preclinical

ST-502 α-Synuclein CNS Preclinical

Undisclosed Neurology CNS Preclinical

TAK-686 Huntington's Disease (HD) CNS PreclinicalTakeda collaboration; Sangamo entitled to

single-digit royalties

Undisclosed ALS/FTD CNS Preclinical

Pfizer collaboration; Sangamo entitled to

up to $150M milestones plus mid- to high-

single digit royalties

Undisclosed Prion CNS Preclinical Wholly-owned

UndisclosedNeurodevelopmental

DisordersCNS Preclinical

Novartis collaboration; Sangamo entitled to

up to $420M development and $300M

commercial milestones

Total pipeline 3,058 3,211 11,522 11,861

Risk-Adjusted Not Risk-Adjusted

1,066 4,613

Ex Vivo

Gene-Edited

Cell Therapy

In Vivo

Genome

Regulation

HCW Peak Sales Estimates

Giroctocogene

FitelparvovecHemophilia A (Hem-A) Liver Phase 3

Gene Therapy

Sanofi collaboration; co-promote option in

U.S.; Sangamo entitled to up to $276.3M

plus tiered double-digit royalties

Biogen collaboration; neurological including

AD and PD; Sangamo entitled to up to

$2.37B ($925M pre-commercial)

milestones plus high-single to sub-teen

double-digit royalties

Kite (Gilead) collaboration; anti-CD19 CAR-

T; Sangamo entitled to up to $3B

milestones plus single digit royalties

Pfizer collaboration; Sangamo entitled to

up to $300M milestones plus 14% to 20%

royalties

1,066 4,613 2030E



Catalyst Calendar


*Clinicaltrials.gov lists August 2022 as primary study completion date.

Compound Indication Milestone Timing Comments Impact

TX200Solid organ transplant

(CAR-Treg)

Phase 1/2 study start

(STEADFAST)

1H21 • Important study start in first-in-class

CAR-Treg program

• Low

ST-400β-Thalassemia Phase 1/2 trial (Thales)

data follow-up readout

By end

of 2021

• High

SAR445136Sickle Cell Disease (SCD) Phase 1/2 trial (PRECIZN-1)

data readout

By end

of 2021

• High

Follow-up data from the Alta

study once all five patients

in the 3e13 vg/kg dose

cohort have been followed

for at least one year

4Q21 • Medium

Pivotal data readout from

Phase 3 trial (AFFINE)

2H22* • Highly anticipated Phase 3 trial data on

Sangamo's lead compound that while

partnered underpins our valuation

• High

ST-920Fabry disease Initial data readout from the

Phase 1/2 trial (STAAR)

End of

2021

• Could provide initial human data in an

important wholly-owned program

• High

• Next key hurdles in programs partnered

with Sanofi; could provide further validation

to the gene editing platform

• Should provide further evidence of

efficacy in potential blockbuster Hem-A

program partnered with Pfizer

Hemophilia A (Hem-A)Giroctocogene

Fitelparvovec



Reiterate $25 Price Target and Buy Rating

• We value Sangamo using a DCF model based on net income per asset and free cash flow to the firm (FCFF), respectively.

The discount rate of 12% in the SOTP and DCF is comparable to clinical stage companies we cover. In our SOTP model, we

value each asset that we assign value to, including SB-525, ST-920, ST-400, and SAR445136, and Sangamo’s ZFP platform

value based on discounted net income, which includes an allocation of operating expenses and taxes to each asset.

• In our DCF, we start with GAAP net income and then add back selected non-cash items to arrive at FCFF for 2021 to 2035.

We think this timeframe extends long enough to capture the value from Sangamo’s pipeline and to reflect competitive

dynamics we anticipate over time. These cash flows are discounted back to approximately one year from now, as is the

present value of the terminal value, which is based on EBITDA in 2035 and a terminal multiple of 6.0x. This compares to the

long-term SMID biopharmaceutical average of approximately 10.5x and the 6.0x we are assuming for other

biopharmaceutical companies in our coverage.

• After adding back cash and securities of approximately $692M and subtracting debt of $38.4M we arrive at an implied equity

value of $3.7B. On diluted shares of approximately 149.1M, Sagnamo’s implied value is approximately $25/share or more

than 125% higher than the current share price.

• Hence, we reiterate our Buy rating on SGMO shares.

• Email us for a copy of our DCF and SOTP models.




DCF and SOTP Model


$ in millions, unless noted


Discount Rate 12.0%

Terminal Value EV/EBITDA Multiple 6.0x

2018A 2019A 2020A 2021E 2022E 2023E 2024E 2025E 2026E 2027E 2028E 2029E 2030E 2031E 2032E 2033E 2034E 2035E

GAAP Net Income (68.3) (95.2) (121.0) (182.3) (271.1) (264.7) (220.0) (24.0) 227.7 548.9 889.4 963.6 1,081.6 1,133.7 976.9 927.8 883.9 880.4

Plus: Depreciation and Amortization 2.4 3.9 5.7 4.1 3.0 8.0 13.0 18.0 23.0 28.0 33.0 38.0 43.0 43.0 43.0 43.0 43.0 43.0

Less: Investment in Working Capital 93.3 (73.0) 252.6 (34.9) 0.0 0.0 (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0) (20.0)

Less: Investment in Fixed Capital (43.1) (20.7) (14.7) (31.8) (33.8) (20.0) (40.0) (42.0) (44.0) (46.0) (48.0) (50.0) (52.0) (54.0) (56.0) (58.0) (60.0) (62.0)

Free Cash Flow to the Firm (FCFF) (15.8) (184.9) 122.6 (244.8) (301.9) (276.7) (267.0) (68.0) 186.7 510.9 854.4 931.6 1,052.6 1,102.7 943.9 892.8 846.9 841.4

Cumulative FCF 7,005.5

EBITDA (2035) 1,198.9

Terminal Value 7,193.4

PV of Free Cash Flow 1,570.5 (244.8) (301.9) (276.7) (267.0) (68.0) 105.9 258.8 386.5 376.2 379.6 355.0 271.3 229.2 194.1 172.2

PV of Terminal Value 1,471.9

Implied Enterprise Value 3,042.4

Plus: Cash and Securities 692.0

Less: Total Debt 38.4 $25 5.5x 6.0x 6.5x

Implied Value of Equity 3,696.0 11.5% $25 $26 $27

Diluted Shares Outstanding 149.1 12.0% $24 $25 $26

Implied Value per Share $25 12.5% $23 $23 $24

Implied Equity Value Sensitivity Table

Terminal Value EV/EBITDA Multiple

Dis

co

un

t Ra

te

$10

$2

$4

$6

$5

$5 $7

$25

$0

$5

$10

$15

$20

$25

$30

$35

SB-525 -Hem-A

ST-400 - β-Thal

SAR445136 - SCD

ST-920 -Fabry

ZFP Platform

Cash and Securities

Unallocated Expenses

Total



ST-920 Could Become Leading Treatment in Fabry

Disease, a Potential $500M-Plus Rare Disease Indication



Brief Background on Selected Viral Gene Therapy Modalities

Source: Company filings, Bulcha, Wang, et al., H.C. Wainwright & Co.

Viral gene therapy modalities. In vivo gene therapy entails the direct administration

of vector carrying a therapeutic transgene into the patient. Ex vivo gene therapy

involves the extraction of a patient’s cells or from an allogenic source, genetic

modification by a vector carrying a therapeutic transgene, selection and expansion in

culture, and infusion to re-introduce the engineered cells back into the patient.

• Gene therapy (GT) is the treatment of a genetic disease by the introduction of

specific cell function-altering genetic material into a patient. The key step in

GT is efficient gene delivery to the target tissue/cells, which is carried out by

gene delivery vehicles called vectors.

• There are two types of vectors: viral and non-viral. Contemporary viral vector-

based gene therapy is achieved by in vivo delivery of the therapeutic gene into

the patient by vectors based on retroviruses, adenoviruses (Ads) or adeno-

associated viruses (AAVs). Alternatively, a therapeutic transgene can be

delivered ex vivo, whereby cells of a patient are extracted and cultured outside

of the body. Cells are then genetically modified by introduction of a therapeutic

transgene and are then re-introduced back into the patient.

• There are four basic gene therapy approaches as follows: gene replacement,

the delivery of a functional gene to replace a non-working gene; gene

silencing, inactivation of a mutated gene that has become toxic to cells; gene

addition, over expression of a “foreign” or exogenous gene to impact cellular

function; and gene editing, a permanent manipulation of a gene in a patient’s

genome.

• AAV vectors have been universally recognized as versatile vectors for gene

therapy. Much of this is owed to their wide-ranging tropism profiles, even by

the contemporary serotypes. As a result, most AAV vector platforms have

been developed to target diseases of the central nervous system, the eyes,

liver, heart, and muscle. The main consideration for AAV vector design is that

the wild-type genome is approximately 4.7 kb in size. Thus, vectors based on

them are irrevocably limited to an approximate 5 kb capacity. Additional

important aspects of vector design: (1) promoters or regulatory cassettes used

in AAV vectors, which drive transgene expression; and (2) capsids, which are

critical for recognition of cell surface proteins that impact tissue and cell

tropism, and also serve as the epitope for antibody recognition.

Summary of viral vectors used in clinical trials. (a) shows a pie chart with the

percentage of adenovirus, adeno-associated virus, or lentivirus vectors in use; (b)

shows a table including the current number of clinical trials employing the different

viral vectors.

Source: Bulcha, Wang, et al., 2021.



Selected Vectors: Advantages, Disadvantages, and Future Directions

Source: Company filings, Nature, H.C. Wainwright & Co.

Vector Ad AAV Lentivirus

Advantages

• Delivery of many genome copies per target cell, that

typically translates into very high expression.

• Relatively large therapeutic genes (25-30 kb with the

latest generation vectors).

• Vector genomes are genetically stable.

• In vivo administration.

• Extensive clinical experience.

• Minimal issues with insertional mutagenesis.

• Several genome copies per target cell.

• Stable expression in some settings.

• In vivo administration.

• Generally safe at doses tested.

• Transduction rates of up to 90% of HSCs.

• High levels of transgene's expression.

• High titers (>108 TU/ml)

• Extensive clinical experience owing to HIV.

• High fidelity gene transfer due to intact integration

and absence of chromosomal rearrangements.

• Reduced genotoxicity vs. gammaretroviral vectors.

Challenges

• Since adenoviruses are non-integrating viruses, the

transgene expression typically lasts 1-2 months in non-

dividing cells, while is much shorter in dividing cells.

• High worldwide prevalence of pre-existing immunity

against common Ad serotypes including HAd5.

• Prevalence rates for NAbs against HAd5 range from

35% in the U.S. to more than 90% in Cote d'lvoire.

• Immunogenicity.

• Cellular toxicity.

• Small transgenes (approximately 4.7 kb)

• Transfer and expression are not always stable.

• Immunogenicity towards the vector remains the

largest challenge for AAV-based gene therapies.

• Managing the right treatment doses, which which

could be the cause of strong immunological responses

and subsequent toxicities seen in recent trials.

• Sensitive to chromosomal position effects.

• Limits on the size of the therapeutic gene (<10 kb)

insert.

• Potential problems with insertional mutagenesis due

to integration inside or near a proto-oncogene.

• Even self-inactivating lentiviral vectors with strong

promoter and enhancer elements can activate

neighboring genes.

• Integration can potentially form chimeric gene fusions

made up of proviral and host sequences.

• Lentiviral vectors have been shown to cause aberrant

splicing of cellular transcripts.

Future

Directions

• Several “rare” human serotypes with low

seroprevalence, such as HAd2, HAd26, and HAd35.

• Various non-human Ad vectors were developed to

limit cross-reactive immunity.

• High-capacity (HCAd) vectors can achieve long-term

transgene expression in vivo, since they have

dampened host immune responses against viral

proteins that may be residually expressed.

• Despite some setbacks, AAV vectors still hold

significant promise for correcting disease.

• Expect improvements to vector design to optimize

transgene expression while minimizing

immunogenicity.

• Continued efforts in guaging the long-term and short-

term safety for AAV vectors.

• Due to their ability to transduce non-dividing cells,

such as dendritic cells, recombinant lentiviral vectors

have been shown to elicit B cell- and T cell-mediated

immunity against infectious diseases and different

tumors.

• Recent advances in the development of non-

integrating lentiviral vectors have greatly reduced

insertional mutagenesis.

• Application of non-integrating lentiviral vectors to

circumvent immune responses mounted against

prolonged expression of genome editing tools, such as

CRISPR/Cas, facilitates the use of such systems in

therapeutic gene editing.



• Fabry disease is a rare, X-linked disease characterized by a congenital error of

glycosphingolipid metabolism caused by an abnormal gene encoding the α-Gal A

enzyme, which breaks down glycosphingolipids. Deficiency of αGLA results in the

accumulation of glycosphingolipids, such as Gb3, and its deacetylated form,

globotriaosylsphingosine, or Lyso-Gb3, that are associated with the

pathophysiology of the disease.

• There are several mutations underlying Fabry disease, which can be divided into

two categories: classic phenotype, the most severe form of the disease, which only

affects men without residual enzyme activity, and a generally milder nonclassic

phenotype also known as the atypical or late-onset form of the disease, which is

suffered by both men and women and is characterized by a more variable disease

course, in which patients are generally less severely affected and disease

manifestations may be limited to a single organ such as the kidneys or the heart.

• Fabry disease is associated with major disability, organ failure and premature

death, generally before the age of 60 years. Late-onset variants present later in life

with organ dysfunction which can be as severe as in the classic form of the

disease, but without the peripheral stigmata of pain, sweating abnormalities and

angiokeratoma. Fabry disease also affects heterozygote females with the defective

gene, where symptoms can develop about a decade later than males.

• Fabry disease is also a pan-ethnic disorder, meaning that it occurs in all racial and

ethnic populations affecting males and females. It is estimated that classic type

Fabry disease affects approximately one in 40,000 males. However, newborn

screening initiatives have suggested an even higher prevalence of the disease

(one in 3,100 male newborns found by a study conducted in Italy).

Source: Company filings, NORD, H.C. Wainwright & Co. Source: NORD.

Brief Background on Fabry Disease



Fabry Disease: Additional Details

Source: Company filings, NORD, H.C. Wainwright & Co.

• Treatments and limitations: ERT. Currently no curative

therapies exist, and alternatives to enzyme replacement

treatment (ERT) for patients with Fabry disease are limited.

Fabry disease is primarily treated with periodic infusions of

ERT consisting of αGLA over the patient’s lifetime. The most

commonly prescribed ERTs for Fabry disease include

Sanofi’s Fabrazyme (agalsidase beta) and Takeda’s Replagal

(agalsidase alpha). ERT is beneficial for patients, reducing

Gb3 substrate levels and extending life expectancy for

patients, though requires chronic every other week infusions

over a life span and over time can lead to antibodies that limit

treatment effectiveness; 40% of male patients with classic

Fabry disease could develop anti-GLA antibodies. Moreover,

because of suboptimal pharmacokinetics and very short half-

life of a few hours, ERTs only temporarily increase plasma

enzyme levels and as a result patients continue to have

disease progression, including ongoing decline in renal

function, potentially resulting in renal failure, cardiovascular

disease and ongoing debilitating pain including periods of

severe pain crisis. ERT is also expensive, costing hundreds

of thousands of dollars annually.

• Treatments and limitations: Chaperone therapy. Amicus’

Galafold (migalastat) was approved by the EMA in May 2016

and FDA in September 2018. Galafold is a pharmacologic

chaperone that can bind to, stabilize, and enhance the

residual enzymatic activity of certain missense mutations.

Amicus reports that 35% to 50% of the gene mutations

associated with Fabry disease are amenable to migalastat,

implying a significant portion of the patient population is

ineligible.

Note: As shown above, therapies are directed at either replacing or generating deficient enzyme,

or blocking the accumulation of substrate. Clinical trials with gene therapy approaches to the

treatment of Fabry disease are ongoing. Chaperone therapy is now available for amenable

mutation with migalastat. Enzyme replacement therapy remains the mainstay of treatment for

most patients with Fabry disease, with agalsidase α and agalsidase β, while pegunigalsidase is

in clinical trials. Substrate reduction therapy (SRT) consists of glucosylceramide synthase

inhibitors and aims to reduce the accumulation of toxic substrate; SRT agents are currently in

clinical trials.

Current and investigational therapeutic agents for Fabry disease at each potential

point of therapeutic intervention.

Source: Kidney Int Rep. 2020 Apr; 5(4): 407–413.



• ST-920. ST-920 is a recombinant adeno-

associated virus (rAAV) 2/6 vector encoding the

cDNA of human α-Gal A (AGA), which has a

sequence identical to the wild type enzyme. The

constant production of AGA in humans should

enable reduction and potentially clearance of

Fabry disease substrates Gb3 and lyso-Gb3 from

target organs. Importantly, we note that the same

rAAV capsid liver-targeted gene therapy has

been previously administered in patients with

hemophilia A (SB-525), demonstrating a positive

risk-benefit profile in data generated to-date.

• Compelling preclinical data. A detailed 3-

month pharmacology and toxicology study

showed that administration of a clinical-scale-

manufactured AAV2/6 vector resulted in

markedly increased plasma and tissue α-Gal A

activities, and essentially normalized Gb3 and

Lyso-Gb3 at key sites of pathology. Further

optimization of vector design identified the clinical

lead vector, ST-920, which produced several-fold

higher plasma and tissue α-Gal A activity levels

with a good safety profile.

Source: Yasuda, Huston, et al., 2020, H.C. Wainwright & Co.

ST-920, Built With Validated Platform, Generated Positive Preclinical DataClinical-scale manufactured ST-920PC/Sf9

vector results in supraphysiological levels of

α-Gal A in plasma and tissues, normalizing

substrates in key tissues in Fabry mice.

Various AAV2/6 hGLA vectors evaluated.

ST-920/Sf9 is more efficacious than ST-

920PC/Sf9 in vivo.

Source: Yasuda, Huston, et al., 2020.



STAAR design.

• Eligibility (screening up to eight weeks). STAAR includes men aged

18 years and older with diagnosis of classical Fabry disease. Patients

are to be enrolled from 10-15 clinics in the U.S. and UK. Patients can

continue on ERT during the trial.

• Key exclusion criteria. Known to be unresponsive to ERT;

neutralizing antibodies to AAV2/6; eGFR ≤ 60 ml/min/1.73m2;

contraindication to use of corticosteroids for immunosuppression; non-

classical Fabry disease.

• Primary endpoint. Incidence of treatment-emergent adverse events

(TEAEs) during 12 months after ST-920 infusion. Additional safety

evaluations are to include routine hematology, chemistry and liver

tests, vital signs, ECG and ECHO, serial alpha fetoprotein (AFP)

testing and liver MRI to monitor for any liver mass formation.

• Secondary endpoints. Change from baseline at specific time points

during the follow-up period: AGA activity in plasma; Gb3 and lyso-GB3

plasma levels; frequency of ERT; estimated glomerular filtration rate

(eGFR) calculated by creatinine levels in blood; and vector clearance

measured by level of vector genome in blood, saliva, urine, stool, and

semen.

• Exploratory endpoints. Left ventricular mass (measured by cardiac

MRI); quality of life; Fabry symptoms and neuropathic pain scores;

immune response to AAV and to AGA.

• Our view. Based on the data generated to-date we believe ST-920

could demonstrate the ability to normalize AGA activity in plasma in

adult males with classical Fabry disease.

Source: Company filings, Sangamo STAAR clinical trial presentation, H.C. Wainwright & Co.

ST-920: Phase 1/2 Trial (STAAR) Should Generate Initial Data By 4Q21



Benchmarking the Competition: Freeline’s FLT190 Appears Promising


• FLT190. Freeline is developing FLT190 for treatment of Fabry disease. FLT190, based on the AAVS3 capsid, could provide sustained levels of functional

αGLA at a lower vector dose than other gene therapy programs for this indication, according to Freeline. To be sure, Freeline’s goal for FLT190 is to

bring αGLA activity in patients into the normal range with sustained steady state levels of between five to nine nmol/hr/ml, and potentially to a level that is

above the normal range, thus providing a treatment for Fabry disease that has the potential to be more efficacious, better tolerated and less burdensome

than ERT. We note that FLT190 builds on experience with FLT180a; both candidates utilize the same manufacturing process, and the capsid and

promoter are identical. As a result, we believe Freeline should be able to take learnings from the HB program, including predicted dose response and

immune management, into the Fabry program to potentially streamline and improve the clinical development program.

• Initial efficacy signal at lowest dose could imply next

doses in escalation phase enable patients to achieve

normal plasma GLA level. As of February 11, 2021, one

patient has been dosed with FLT190 in MARVEL-1. The first

patient was administered FLT190 at a dose of 7.5e11 vg/kg;

the infusion has been generally well-tolerated with no infusion-

related reaction, and no dose-limiting toxicity events (DLTs)

have been observed. Plasma GLA activity rose from a

baseline trough of 0.3 nmol/hr/ml to 1.2 nmol/hr/ml by week

four.

• Stable, elevated GLA expression after one year. The first

patient dosed continues to show stable elevated GLA

expression at 1 nmol/hr/cc at 12 months post treatment which

is approximately a 3-fold increase compared to their baseline

level. The subject restarted ERT from week six as the GLA

levels remained below the lower threshold of normal and

some peripheral nerve-related symptoms were reported.

Based on the dose response seen in B-AMAZE, a Phase 1/2

dose finding trial in hemophilia B evaluating FLT180a,

Freeline believes a dose of either 1.5e12 or 4.5e12 vg/kg has

the potential to bring patients into, or above, the normal range

of GLA expression.

FLT190 produced a 3-fold increase in plasma GLA, sustained over one year post-

treatment

Note: Data as of June 15, 2020; normal range for GLA in plasma is 5-9 nmol/hr/ml; below 1 nmol/hr/ml is

diagnostic for Fabry disease.

Source: Freeline corporate presentation.



• AVROBIO. Lead candidate, AVR-RD-01, is derived from hematopoietic

stem cells to which the gene encoding plasma AGA is added in an ex vivo

process using a lentiviral vector.

• Positive data updates. In February 2021, AVROBIO reported that a kidney

biopsy taken at 12 months post-treatment from the fourth patient in the

Phase 2 trial (FAB-GT), who was the first in the trial dosed with AVROBIO’s

“plato” platform, showed a 100% reduction from baseline in the average

number of Gb3 inclusions per peritubular capillary (PTC). In addition, in

February 2021, AVROBIO reported data from the first four patients in the

FAB-GT trial measured as of 29, 24, 18 and 12 months, respectively,

indicating sustained increased leukocyte and plasma enzyme activity,

suggesting that these patients are now producing an endogenous supply of

AGA, as well as decreased plasma lyso-Gb3 levels in three of four patients.

These data followed a 2019 announcement that at 12 months post-

treatment the first patient in the FAB-GT clinical trial exhibited an 87%

reduction in the average number of Gb3 inclusions per PTC as compared to

baseline.

• Safety and tolerability. Preliminary interim safety data from Phase 1 and 2

trials indicate AVR-RD-01 has been generally well-tolerated with no SAEs

related to the study drug. Grade 3 or 4 AEs have generally been attributed

to the melphalan conditioning regimen prescribed by the study protocol.

• Adverse regulatory update. On May 3, AVRO announced it can no longer

pursue an accelerated approval with a Phase 2 trial (FAB-GT) and instead

intends to discuss with FDA a registration trial with a primary efficacy

endpoint of clearance of GL-3 (also referred to as Gb3) inclusions in

biopsied renal peritubular capillaries (PTCs) as the basis for full approval.

AVRO noted that on March 11, approximately three weeks before a planned

End-of-Phase 1 meeting, FDA granted full approval of Fabrazyme

(agalsidase beta), an enzyme replacement therapy (ERT), more than 18

years after it received accelerated approval on the basis of a surrogate

endpoint: reduction of GL-3/Gb3 inclusions in biopsied renal PTCs.


Benchmarking the Competition: AVROBIO’s AVR-RD-01

Patient 1 Patient 4

Source: AVROBIO corporate presentation.

Phase 2 trial

70% average plasma lyso-Gb3 reduction

Source: AVROBIO corporate presentation.

Phase 2 FAB-GT: Patients 1 and 4 substrate reduction in kidney biopsy



• Amicus. Amicus’ orally administered precision medicine monotherapy, Galafold, is designed to bind to and stabilize an endogenous αGA

enzyme in those patients with genetic variants identified as amenable in a GLP cell-based amenability assay. Amicus believes 35-50% of

Fabry disease patients may benefit from treatment with Galafold. Given the aforementioned advantages of GT treatment, to us, the

competition from Amicus vis a vis ST-920 comes more so from its GT candidate. In February 2021, Amicus presented initial preclinical

data from its investigational AAV gene therapy program, in development in collaboration with Penn. The initial preclinical study assessed a

range of single doses of AAV in Gla knockout mice with either wild-type hGLA (unmodified hGLA) or an Amicus/Penn engineered hGLA

transgenes (engineered hGLA or AT-GTX-701). The engineered hGLA AAV gene therapy demonstrated stable homodimer formation,

enhanced temperature, plasma and neutral ph stability compared to the unmodified hGLA AAV gene therapy. In the lowest tested dose of

AT-GTX-701, Gla knockout mice showed partial substrate reduction, while highest tested dose resulted in near complete substrate

reduction. Additionally, AT-GTX-701 demonstrated significantly greater lyso-Gb3/GL-3 substrate reduction across all Fabry disease

relevant tissues including the dorsal root ganglia (DRG), kidney, and heart, with reductions at low dose being equal to or greater than the

reductions observed at higher doses with wild-type transgene and provided the first evidence for DRG storage reduction in a Fabry mouse

model treated with an AAV gene therapy according to Amicus.

• uniQure. In September 2020, uniQure selected a lead gene therapy candidate (AMT-190) for the treatment of Fabry disease to advance

into IND-enabling studies. The lead candidate is a one-time administered AAV5 gene therapy incorporating the GLA transgene. In

preclinical studies comparing multiple product candidates, including constructs incorporating a modified alpha-N-acetylgalactosaminidase

transgene (modNAGA), AMT-190 demonstrated the most robust and sustained increases in GLA activity according to uniQure.

• Our view. We anticipate a robust market for GTs to emerge in Fabry over the next 10 years and believe ST-920 should vie for a leading

share of the market based on the preclinical data generated to date and the positive read through to the platform based on compelling

data generated to date on Sangamo’s most advanced compound, SB-525, partnered with Pfizer and in Phase 3 development (AFFINE).


Benchmarking the Competition: Additional Early Stage GTs



Source: Company filings, Evaluate Pharma, H.C. Wainwright & Co.

Economic Case for GT in Fabry Disease Appears Clear to Us

Consensus estimates suggest ERT and chaperone

treatments could increase from $1.8B globally in 2020 to

$2.7B in 2026, a 50% increase.

If we assume a Fabry patient could receive SOC for 50 years, at

branded pricing, the global cost is near $120B, all else being

equal. Even if we assume biosimilars and generics enter the

market at a 50% and 80% reduction in cost, respectively, the

potential lifetime cost could still be more than $50B for SOC.

$ in millions

Product Company 2020A 2021E 2022E 2023E 2024E 2025E 2026E 5-year 50-year (Rx) 50-year (Gx)

Fabrazyme Sanofi

(SNY; not rated)

933 988 1,015 1,040 1,064 1,087 1,101 5,307 53,070 26,535

Galafold Amicus Therapeutics

(FOLD; not rated)

261 313 386 461 513 567 624 2,551 25,510 5,102

AVR-RD-01 AVROBIO

(AVRO; not rated)

0 0 20 138 234 268 457 1,117 11,170 5,585

Replagal Takeda

(TAK; not rated)

483 500 506 462 433 413 399 2,213 22,130 11,065

FLT190 Freeline

(FRLN; Buy)

0 0 0 0 0 88 248 336 3,360 1,680

Lucerastat Idorsia

(IDRSF; not rated)

0 0 13 61 116 161 202 553 5,530 2,765

Lucerastat Undisclosed

Partner

0 0 8 55 129 176 193 561 5,610 2,805

Replagal Sumitomo Dainippon

Pharma (Japan)

127 129 126 122 122 121 121 612 6,120 3,060

ST-920 Sangamo Therapeutics

(SGMO; Buy)

0 0 0 0 9 36 84 129 1,290 645

Agalsidase Beta

BS

JCR Pharmaceuticals

(Japan)

7 12 16 19 22 25 27 109 1,090 545

ERT + Chaperone

Subtotal

1,811 1,942 2,070 2,220 2,399 2,550 2,667 11,906 119,060 51,877

Total 1,811 1,942 2,090 2,358 2,642 2,942 3,456 13,488 134,880 59,787

Aggregate



Fabry disease market (U.S.).

• We model a potential patient population of around 4,000 in 2022 that expands to 6,000 by 2035.

Eligible patients.

• AVR-RD-01: we assume no exclusions for NAb status.

• FLT190: we assume 50% of patients are eligible.

• ST-920: we assume 65% of patients are eligible.

• AAV: we assume 65% of patients are eligible.

• AMT-190: we assume 95% of patients are eligible.

Penetration rate.

• AVR-RD-01: we assume a potential launch in 2023 with a 1% share that expands to 15% at peak.

• FLT190: we assume a potential launch in 2024 with a 1% share that expands to 20% at peak.

• ST-920: we assume a potential launch in 2024 with a 1% share that expands to 20% at peak.

• AAV: we assume a potential launch in 2025 with a 1% share that expands to 10% at peak.

• AMT-190: we assume a potential launch in 2026 with a 1% share that expands to 10% at peak.

Probability of success (POS).

• We assume 40% for FLT190 and 45% for ST-920 based on the preclinical and clinical data generated to-date.

• We do not yet include AMT-190 in our uniQure model.

Pricing.

• For now we assume all GTs are priced at $3M per treatment, with a 5% reduction for rebates and discounts to payors. The increased number of

competitors could lead to a lower price point relative to HB, in our view, all else being equal.

Loss of exclusivity (LOE).

• We do not model a LOE per se; however, as noted, the eligible patient population is expected to decline as patients are treated with gene therapies.

Source: Company filings and presentations, H.C. Wainwright & Co.

Market Model Assumptions: GT in Fabry Disease



0%

5%

10%

15%

20%

25%

AVR-RD-01 (AVRO) FLT190 (FRLN) ST-920 (SGMO) AAV (FOLD) AMT-190 (QURE)

Source: H.C. Wainwright & Co. estimates.

Market Model Assumptions: GT in Fabry

Of the compounds in development we believe

FLT190 and ST-920 could end up being the most

competitive given the advantages of the AAV

approach (non-integrating), and the safety and

efficacy demonstrated to-date. AAV and AMT-190

are behind the other programs, which is why we

model a lower share, at this time.

The market for GT is still wide open in Fabry, in

our view. For now, we are assuming parity pricing

among the many potential competing GT

treatments, if approved, at $3M per treatment,

before payments of discounts and rebates to

payors.

Estimated penetration rate by year

Estimated cost by year

Note: in the above Exhibit, we assume all treatments are priced at parity to each other. Prices

above are after potential discounts and rebates to payors.

$0

$500,000

$1,000,000

$1,500,000

$2,000,000

$2,500,000

$3,000,000




0

200

400

600

800

1,000

1,200

1,400

1,600

1,800

2022E 2023E 2024E 2025E 2026E 2027E 2028E 2029E 2030E 2031E 2032E 2033E 2034E 2035E

AVR-RD-01 (AVRO) FLT190 (FRLN) ST-920 (SGMO) AAV (FOLD) AMT-190 (QURE) 0

2,000

4,000

6,000

8,000

10,000

12,000

14,000

AVR-RD-01 (AVRO)

FLT190 (FRLN) ST-920 (SGMO) AAV (FOLD) AMT-190 (QURE)

Selected Fabry disease Gene

Therapies

0

5,000

10,000

15,000

20,000

25,000

AVR-RD-01 (AVRO)

FLT190 (FRLN) ST-920 (SGMO) AAV (FOLD) AMT-190 (QURE)

Selected Fabry disease Gene

Therapies


Global Fabry Disease GT Market Could Exceed $30B$ in millions, unless noted $ in millions, unless noted

$ in millions, unless noted $ in millions, unless noted


Estimated per year U.S. revenuesEstimated aggregate U.S. revenues

Estimated aggregate rest-of-world revenuesEstimated per year rest-of-world revenues

0

500

1,000

1,500

2,000

2,500

3,000

3,500

2022E 2023E 2024E 2025E 2026E 2027E 2028E 2029E 2030E 2031E 2032E 2033E 2034E 2035E




Public Companies Mentioned in This Report

Amicus Therapeutics (FOLD; not rated)

AVROBIO (AVRO; not rated)

Freeline (FRLN; Buy; Trucchio)

Pfizer (PFE; not rated)

Sanofi (SNY; not rated)

Takeda (TAK; not rated)

uniQure (QURE; Buy; Trucchio)

Source: H.C. Wainwright & Co.



$ in millions, unless otherwise noted

2018A 2019A 1Q20A 2Q20A 3Q20A 4Q20A 2020A 1Q21A 2Q21E 3Q21E 4Q21E 2021E 2022E

Revenues 84.5 102.4 13.1 21.6 57.8 25.8 118.2 26.3 32.5 32.5 32.5 123.8 78.8

% chg 21.3% 62.0% 22.8% 163.1% -53.0% 15.4% 101.0% 50.8% -43.7% 26.0% 4.7% -36.4%

Cost of goods sold 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Gross profit 84.5 102.4 13.1 21.6 57.8 25.8 118.2 26.3 32.5 32.5 32.5 123.8 78.8

% chg 21.3% 62.0% NM 163.1% -53.0% 15.4% 101.0% 50.8% -43.7% 26.0% 4.7% -36.4%

% of sales NM NM NM NM NM NM NM NM NM NM NM NM

bps chg NM NM NM NM NM NM NM NM NM NM NM NM

Total R&D expenses 114.9 145.9 41.5 41.5 45.3 52.4 180.6 56.4 57.9 59.4 60.9 234.7 256.7

% chg 27.0% 19.0% 13.9% 24.8% 36.6% 23.8% 36.1% 39.5% 31.2% 16.4% 29.9% 9.4%


SG&A expenses 46.7 61.7 16.1 17.9 16.2 16.9 67.1 16.1 17.6 19.1 20.6 73.6 95.6

% chg 32.0% -5.8% 22.8% 8.4% 12.1% 8.8% 0.2% -1.6% 18.4% 22.4% 9.7% 29.9%


Total operating expenses 161.6 207.6 57.6 59.5 61.5 69.2 247.7 72.6 75.6 78.6 81.6 308.3 352.3

% chg 28.5% 10.8% 16.4% 20.0% 29.7% 19.3% 26.0% 27.1% 27.9% 17.8% 24.5% 14.3%


Operating income (loss) (77.2) (105.2) (44.5) (37.9) (3.7) (43.4) (129.6) (46.3) (43.1) (46.1) (49.1) (184.5) (273.6)

% chg NM NM NM NM NM NM NM NM NM NM NM NM


bps chg NM NM NM NM NM NM NM NM NM NM NM NM

Depreciation and amortization 2.4 3.9 1.3 1.3 1.4 1.7 5.7 1.9 0.8 0.8 0.8 4.1 3.0

GAAP EBITDA (74.8) (101.3) (43.2) (36.6) (2.3) (41.7) (123.9) (44.4) (42.3) (45.3) (48.3) (180.4) (270.6)



Total other income, net 8.3 9.8 1.5 1.9 2.4 2.9 8.8 0.6 0.6 0.6 0.6 2.5 2.5

Pretax income (loss) (68.9) (95.4) (43.0) (36.0) (1.271) (40.6) (120.8) (45.7) (42.5) (45.5) (48.5) (182.0) (271.1)



Income taxes 0.0 0.0 0.0 0.0 0.2 0.1 0.3 0.3 0.0 0.0 0.0 0.3 0.0

Tax rate NM 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0% 0.0%

Non-controlling interest 0.6 0.2 0.1 0.0 (0.0) 0.1 0.1 0.0 0.0 0.0 0.0 0.0 0.0

GAAP net income (loss) (68.3) (95.2) (42.9) (35.9) (1.6) (40.6) (121.0) (45.9) (42.5) (45.5) (48.5) (182.3) (271.1)



GAAP EPS ($0.70) ($0.85) ($0.37) ($0.26) ($0.01) ($0.29) ($0.90) ($0.32) ($0.29) ($0.31) ($0.32) ($1.25) ($1.76)

% chg NM NM NM NM NM NM NM NM NM NM NM NM NM

Shares outstanding 96.9 112.1 116.1 139.0 143.0 141.5 134.4 143.1 145.1 147.1 149.1 146.1 154.1

Source: Company filings, H.C. Wainwright & Co. estimates





Net income (loss) (68.9) (95.4) (43.0) (36.0) (1.5) (40.7) (121.1) (45.9) (42.5) (45.5) (48.5) (182.3) (271.1)

Depreciation and amortization 2.4 3.9 1.3 1.3 1.4 1.7 5.7 1.9 0.8 0.8 0.8 4.1 3.0

Amortization of (discount) premium on securities (5.8) (4.7) (0.7) (0.4) (0.2) 0.5 (0.8) 0.8 0.0 0.0 0.0 0.8 0.0

Amortization right-of-use assets 0.0 5.7 1.9 1.9 1.9 2.0 7.7 2.0 0.0 0.0 0.0 2.0 0.0

Loss (gain) on free shares 0.0 (0.5) 0.1 (0.1) 0.0 (0.0) (0.1) 0.0 0.0 0.0 0.0 0.0 0.0

Net loss on disposal of property and equipment 0.0 0.1 0.0 0.0 0.2 0.0 0.2 0.0 0.0 0.0 0.0 0.0 0.0

Foreign currency transaction losses, net 0.6 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Stock-based compensation expense 14.7 19.3 5.6 6.8 6.7 6.6 25.7 7.5 7.7 7.9 8.1 31.3 34.5

Loss on lease termination 0.0 0.2 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Other 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Build-to-suit leases 1.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Changes in working capital

Interest receivable (0.1) (0.3) (0.2) 0.4 (0.3) (0.2) (0.4) 0.1 0.0 0.0 0.0 0.1 0.0

Accounts receivable (1.3) (32.2) 29.9 1.4 (33.9) 34.3 31.7 (1.5) 0.0 0.0 0.0 (1.5) 0.0

Inventories 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Prepaid expenses and other current assets (2.8) (6.7) (0.1) (7.7) (1.3) (1.3) (10.4) (1.7) 0.0 0.0 0.0 (1.7) 0.0

Operating lease right-of-use assets 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Prepaid rent 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Accounts payable and accrued liabilities (6.4) (4.2) 0.3 1.2 (0.2) 9.4 10.7 (3.8) 0.0 0.0 0.0 (3.8) 0.0

Accrued compensation and benefits 2.6 4.1 (5.0) 4.7 4.4 2.7 6.9 (7.6) 0.0 0.0 0.0 (7.6) 0.0

Non-current liabilities 2.0 3.7 0.2 0.2 0.2 0.8 1.3 0.8 0.0 0.0 0.0 0.8 0.0

Long-term portion of lease liabilities 0.0 (1.8) (0.9) (0.9) (1.0) (1.0) (3.8) (1.0) 0.0 0.0 0.0 (1.0) 0.0

Deferred revenues 99.4 (35.7) (8.3) 187.2 56.7 (19.0) 216.5 (20.0) 0.0 0.0 0.0 (20.0) 0.0

Total changes in working capital 93.3 (73.0) 15.9 186.5 24.6 25.6 252.6 (34.9) 0.0 0.0 0.0 (34.9) 0.0

Cash from operating activities 37.2 (144.4) (18.9) 160.0 33.1 (4.3) 169.9 (68.6) (34.0) (36.8) (39.6) (178.9) (233.6)

Purchases of marketable securities (451.2) (443.7) (43.6) 0.0 (291.4) (235.8) (570.8) (97.9) 0.0 0.0 0.0 (97.9) 0.0

Acquisition of TxCell, net (75.6) 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Maturities of marketable securities 391.8 404.8 71.1 80.8 53.2 109.5 314.6 144.4 50.0 50.0 50.0 294.4 200.0

Purchase of property and equipment (43.1) (20.7) (3.8) (2.9) (4.0) (4.0) (14.7) (8.0) (8.0) (8.0) (8.0) (31.8) (33.8)

Purchase of Sangamo France shares 0.0 (0.3) 0.0 (0.2) (0.3) (0.2) (0.7) (0.1) 0.0 0.0 0.0 (0.1) 0.0

Sales of marketable securities 0.0 0.0 0.0 0.0 0.0 0.0 0.0 6.9 0.0 0.0 0.0 6.9 0.0

Other investments 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Cash from investing activities (178.1) (59.8) 23.7 77.6 (242.5) (130.5) (271.6) 45.3 42.1 42.1 42.1 171.4 166.2

Proceeds, public offering of stock 215.8 136.3 0.0 0.0 0.0 0.0 0.0 15.7 0.0 0.0 0.0 15.7 0.0

Proceeds, issuance of common stock, Biogen 0.0 0.0 0.0 142.5 0.0 0.0 142.5 0.0 0.0 0.0 0.0 0.0 0.0

Taxes paid, settlement of equity awards (0.3) (0.4) (0.4) (0.0) (0.1) (0.2) (0.8) (2.2) 0.0 0.0 0.0 (2.2) 0.0

Proceeds, issuance of stock, ESOP 0.0 2.0 0.8 0.4 0.0 0.8 2.0 1.8 0.0 0.0 0.0 1.8 0.0

Proceeds, issuance of restricted stock, options 0.0 4.1 0.0 1.3 1.7 6.3 9.3 0.0 0.0 0.0 0.0 0.0 0.0

Proceeds, issuance of common stock 16.2 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Cash from financing activities 231.7 142.0 0.4 144.2 1.6 6.9 153.1 15.2 0.0 0.0 0.0 15.2 0.0

Effect of exchange rates (0.2) 0.2 0.1 (0.1) (0.2) (0.2) (0.4) (0.3) 0.0 0.0 0.0 (0.3) 0.0

Net increase in cash and equivalents 90.6 (62.0) 5.3 381.7 (208.1) (128.1) 50.9 (8.4) 8.1 5.3 2.5 7.5 (67.4)

Beginning cash and equivalents 53.3 143.9 80.4 85.7 467.4 259.4 80.4 131.3 123.0 131.0 136.3 131.3 138.8

Less: Restricted cash 3.5 1.5 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Ending cash and equivalents 140.4 80.4 85.7 467.4 259.4 131.3 131.3 123.0 131.0 136.3 138.8 138.8 71.4

Free cash flow (5.9) (165.1) (22.7) 157.1 29.1 (8.3) 155.2 (76.5) (41.9) (44.7) (47.5) (210.7) (267.4)

FCF/share ($0.06) ($1.47) ($0.20) $1.13 $0.20 ($0.06) $1.15 ($0.53) ($0.29) ($0.30) ($0.32) ($1.44) ($1.73)






Cash and cash equivalents 140.4 80.4 85.7 467.4 259.4 131.3 131.3 123.0 131.0 136.3 138.8 138.8 71.4

Restricted cash 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Marketable securities 259.7 282.0 272.4 197.4 389.4 510.1 510.1 464.1 414.1 364.1 314.1 314.1 114.1

Interest receivable 0.4 0.7 0.9 0.5 0.8 1.0 1.0 1.0 1.0 1.0 1.0 1.0 1.0

Accounts receivable 4.7 36.9 7.0 5.6 39.5 5.2 5.2 6.7 6.7 6.7 6.7 6.7 6.7

Inventory 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Prepaid expenses and other 5.3 5.4 4.9 8.4 11.2 12.0 12.0 12.1 12.1 12.1 12.1 12.1 12.1

Total current assets 410.5 405.5 370.9 679.4 700.3 659.7 659.7 606.9 565.0 520.3 472.8 472.8 205.4

Marketable securities 0.0 21.8 5.0 0.0 45.8 50.5 50.5 42.4 42.4 42.4 42.4 42.4 42.4

Property and equipment, net 78.7 29.9 31.3 33.9 35.3 41.3 41.3 45.2 52.4 59.6 66.8 66.8 97.6

Intangible assets 54.9 53.2 52.1 53.2 55.6 58.1 58.1 55.6 55.6 55.6 55.6 55.6 55.6

Goodwill 40.0 39.3 38.6 39.3 41.0 42.8 42.8 41.0 41.0 41.0 41.0 41.0 41.0

Operating lease right-of-use assets 0.0 77.3 75.4 73.5 71.6 71.0 71.0 70.3 70.3 70.3 70.3 70.3 70.3

Prepaid rent 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Other non-current assets 2.7 9.1 9.5 13.8 12.7 13.6 13.6 14.1 14.1 14.1 14.1 14.1 14.1

Non-current restricted cash 3.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5 1.5

Total assets 590.4 637.5 584.3 894.7 963.7 938.6 938.6 877.1 842.4 804.8 764.5 764.5 528.0

Accounts payable and accrued liabilities 21.5 17.6 16.9 19.2 17.9 12.6 12.6 11.6 11.6 11.6 11.6 11.6 11.6

Other accrued liabilities 0.0 0.0 0.0 0.0 0.0 18.6 18.6 13.8 13.8 13.8 13.8 13.8 13.8

Accrued compensation and employee benefits 9.5 13.6 8.6 13.3 17.9 20.7 20.7 13.1 13.1 13.1 13.1 13.1 13.1

Deferred revenue 47.6 38.7 36.6 61.4 87.8 91.6 91.6 94.4 94.4 94.4 94.4 94.4 94.4

Total current liabilities 78.5 69.9 62.1 93.9 123.6 143.5 143.5 132.8 132.8 132.8 132.8 132.8 132.8

Deferred revenues 108.3 81.4 75.3 237.6 267.9 245.0 245.0 222.3 222.3 222.3 222.3 222.3 222.3

Lease liabilities 27.7 41.2 40.2 39.2 38.3 38.4 38.4 38.5 38.5 38.5 38.5 38.5 38.5

Deferred income tax 6.7 6.6 6.4 6.6 6.9 7.2 7.2 6.9 6.9 6.9 6.9 6.9 6.9

Other non-current liabilities 2.0 5.7 5.9 6.1 6.2 7.0 7.0 7.2 7.2 7.2 7.2 7.2 7.2

Total liabilities 223.1 204.8 189.9 383.5 442.8 441.2 441.2 407.7 407.7 407.7 407.7 407.7 407.7

Preferred stock 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0 0.0

Common stock 1.0 1.2 1.2 1.4 1.4 1.4 1.4 1.4 1.4 1.4 1.4 1.4 1.4

Additional paid-in capital 929.6 1,090.8 1,096.9 1,247.5 1,255.8 1,269.4 1,269.4 1,292.1 1,299.8 1,307.8 1,315.9 1,315.9 1,350.4

Accumulated deficit (562.7) (657.0) (699.9) (735.8) (737.4) (778.0) (778.0) (823.9) (866.4) (911.8) (960.3) (960.3) (1,231.3)

Accumulated other comprehensive income (loss) (1.4) (2.4) (3.8) (1.7) 1.6 5.4 5.4 0.7 0.7 0.7 0.7 0.7 0.7

Non-controlling interest 0.7 0.2 0.1 (0.3) (0.5) (0.9) (0.9) (0.9) (0.9) (0.9) (0.9) (0.9) (0.9)

Total stockholders' equity (deficit) 367.3 432.7 394.4 511.2 520.9 497.4 497.4 469.4 434.7 397.2 356.8 356.8 120.3

Total liabilities and equity 590.4 637.5 584.3 894.7 963.7 938.6 938.6 877.1 842.4 804.8 764.5 764.5 528.0




Important Disclaimers

This material is confidential and intended for use by Institutional Accounts as defined in FINRA Rule 4512(c). It may also beprivileged or otherwise protected by work product immunity or other legal rules. If you have received it by mistake, please letus know by e-mail reply to [email protected] and delete it from your system; you may not copy this message ordisclose its contents to anyone. The integrity and security of this message cannot be guaranteed on the Internet.H.C. WAINWRIGHT & CO, LLC RATING SYSTEM: H.C. Wainwright employs a three tier rating system for evaluating boththe potential return and risk associated with owning common equity shares of rated firms. The expected return of any givenequity is measured on a RELATIVE basis of other companies in the same sector. The price objective is calculated to estimatethe potential movements in price that a given equity could reach provided certain targets are met over a defined time horizon.Price objectives are subject to external factors including industry events and market volatility.

RETURN ASSESSMENTMarket Outperform (Buy): The common stock of the company is expected to outperform a passive index comprised of all thecommon stock of companies within the same sector.Market Perform (Neutral): The common stock of the company is expected to mimic the performance of a passive indexcomprised of all the common stock of companies within the same sector.Market Underperform (Sell): The common stock of the company is expected to underperform a passive index comprised ofall the common stock of companies within the same sector.

Rating and Price Target History for: Sangamo Therapeutics, Inc. (SGMO-US) as of 05-18-2021

201816141210864

Q1 Q2 Q3 2019 Q1 Q2 Q3 2020 Q1 Q2 Q3 2021 Q1 Q2

I:BUY:$16.0008/26/19

BUY:$14.0006/22/20

UR:NA09/22/20

BUY:$25.0012/16/20

Rating and Price Target History for: Amicus Therapeutics, Inc (FOLD-US) as of 05-18-2021

30

25

20

15

10

5Q1 Q2 Q3 2019 Q1 Q2 Q3 2020 Q1 Q2 Q3 2021 Q1 Q2

I:BUY:$18.0006/17/19

BUY:$20.0011/12/19

BUY:$20.0011/21/19

UR:NA09/22/20

Rating and Price Target History for: Freeline Therapeutics Holdings plc (FRLN-US) as of 05-18-2021

20

18

16

14

12

10

8Q1 Q2 Q3 2019 Q1 Q2 Q3 2020 Q1 Q2 Q3 2021 Q1 Q2



Rating and Price Target History for: uniQure N.V. (QURE-US) as of 05-18-2021

90

80

70

60

50

40

30

20Q1 Q2 Q3 2019 Q1 Q2 Q3 2020 Q1 Q2 Q3 2021 Q1 Q2

BUY:$35.0002/20/18

BUY:$42.0005/22/18

BUY:$48.0009/24/18

BUY:$60.0011/26/18

BUY:$73.0002/11/19

BUY:$64.0006/25/20

UR:NA09/22/20

BUY:$80.0011/24/20

BUY:$85.0012/21/20

BUY:$80.0012/22/20



Related Companies Mentioned in this Report as of May/18/2021

Company Ticker H.C. Wainwright 12 Month Price MarketRating Price Target Cap

Freeline Therapeutics Holdings plc FRLN Buy $30.00 $8.42uniQure N.V. QURE Buy $80.00 $32.57 $1499Amicus Therapeutics, Inc FOLD Under Review $NA $9.24

Investment Banking Services include, but are not limited to, acting as a manager/co-manager in the underwriting or placementof securities, acting as financial advisor, and/or providing corporate finance or capital markets-related services to a companyor one of its affiliates or subsidiaries within the past 12 months.

Distribution of Ratings Table as of May 18, 2021IB Service/Past 12 Months

Ratings Count Percent Count PercentBuy 466 89.96% 192 41.20%Neutral 50 9.65% 13 26.00%Sell 0 0.00% 0 0.00%Under Review 2 0.39% 1 50.00%

H.C. Wainwright & Co, LLC (the “Firm”) is a member of FINRA and SIPC and a registered U.S. Broker-Dealer.

I, Patrick R. Trucchio, CFA , certify that 1) all of the views expressed in this report accurately reflect my personal views aboutany and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectlyrelated to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members ofmy household is an officer, director or advisory board member of these companies.

None of the research analysts or the research analyst’s household has a financial interest in the securities of SangamoTherapeutics, Inc., Amicus Therapeutics, Inc and Freeline Therapeutics Holdings plc (including, without limitation, any option,right, warrant, future, long or short position).As of April 30, 2021 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities ofSangamo Therapeutics, Inc., Amicus Therapeutics, Inc and Freeline Therapeutics Holdings plc.Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the timeof publication of this research report.

A research analyst of the firm and/or the research analyst’s household has a financial interest in and own the securities ofuniQure N.V. (including, without limitation, any option, right, warrant, future, long or short position).

As of April 30, 2021 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securitiesof uniQure N.V..Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the timeof publication of this research report.

The research analyst principally responsible for preparation of the report does not receive compensation that is based upon anyspecific investment banking services or transaction but is compensated based on factors including total revenue and profitabilityof the Firm, a substantial portion of which is derived from investment banking services.

The firm or its affiliates received compensation from Sangamo Therapeutics, Inc. and uniQure N.V. for non-investment bankingservices in the previous 12 months.

The Firm or its affiliates did not receive compensation from Sangamo Therapeutics, Inc., Amicus Therapeutics, Inc, FreelineTherapeutics Holdings plc and uniQure N.V. for investment banking services within twelve months before, but will seekcompensation from the companies mentioned in this report for investment banking services within three months followingpublication of the research report.

The Firm does not make a market in Sangamo Therapeutics, Inc., Amicus Therapeutics, Inc, Freeline Therapeutics Holdingsplc and uniQure N.V. as of the date of this research report.

The securities of the company discussed in this report may be unsuitable for investors depending on their specific investmentobjectives and financial position. Past performance is no guarantee of future results. This report is offered for informationalpurposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdictionwhere such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice toany person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously.



No part of this report may be reproduced in any form without the expressed permission of H.C. Wainwright & Co., LLC. Additionalinformation available upon request.H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report isnot intended to provide personal investment advice and it does not take into account the specific investment objectives, financialsituation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriatenessof investing in financial instruments and implementing investment strategies discussed or recommended in this research report.H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written marketcommentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report.H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to timehave long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants)thereof of covered companies referred to in this research report.

The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as beingaccurate and does not purport to be a complete statement or summary of the available data on the company, industry or securitydiscussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date ofthis report and are subject to change without notice.

Securities and other financial instruments discussed in this research report: may lose value; are not insured by the FederalDeposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.



Rating: Buy Sangamo Therapeutics, Inc. (SGMO) 2021 ...

Documents

Transcript of Rating: Buy Sangamo Therapeutics, Inc. (SGMO) 2021 ...