​

(Unaudited)

​

​

(in thousands, except share data)

​

June 30, 2022

​

December 31, 2021

Assets

​

​

Current assets:

​

​

Cash and cash equivalents

​

$

91,129

​

$

128,965

Marketable securities

​

​

148,121

​

​

186,808

Prepaid expenses and other current assets

​

2,316

​

1,726

Prepaid research and development

​

9,011

​

7,567

Total current assets

​

250,577

​

325,066

Property and equipment, net

​

23,566

​

23,806

Right of use assets - operating leases

​

​

20,053

​

​

-

Other assets

​

5,220

​

6,204

Total assets

​

$

299,416

​

$

355,076

Liabilities and stockholders’ equity

​

​

​

Current liabilities:

​

​

​

Accounts payable

​

$

8,211

​

$

9,448

Accrued expenses and other current liabilities

​

15,692

​

20,050

Operating lease liabilities

​

​

3,230

​

​

-

Total current liabilities

​

27,133

​

29,498

Operating lease liabilities - noncurrent

​

24,237

​

-

Deferred rent

​

-

​

6,921

Total liabilities

​

51,370

​

36,419

​

​

​

​

​

​

​

Commitments and Contingencies

​

​

​

​

​

​

​

​

​

​

Stockholders’ equity:

​

​

​

Common stock, $0.0001 par value: 300,000,000 shares authorized; 54,463,235 shares issued and outstanding at June 30, 2022 and 54,244,996 shares issued and outstanding at December 31, 2021

​

5

​

5

Additional paid-in capital

​

688,253

​

675,346

Accumulated other comprehensive income (loss)

​

​

(1,578)

​

​

(413)

Accumulated deficit

​

(438,634)

​

(356,281)

Total stockholders’ equity

​

248,046

​

318,657

Total liabilities and stockholders’ equity

​

$

299,416

​

$

355,076

​

​

Three Months Ended June 30, 

​

Six Months Ended June 30, 

(in thousands, except share and per share data)

2022

2021

​

2022

2021

Operating expenses:

​

​

​

​

​

Research and development

​

$

26,821

​

$

33,112

​

$

53,034

​

$

58,082

Acquired in-process research and development

​

—

​

—

​

1,500

​

1,500

General and administrative

​

12,991

​

15,422

​

28,090

​

27,886

Loss from operations

​

(39,812)

​

(48,534)

​

(82,624)

​

(87,468)

Interest income, net

​

270

​

99

​

271

​

151

Net loss

​

$

(39,542)

​

$

(48,435)

​

$

(82,353)

​

$

(87,317)

Per share information:

​

​

​

​

​

Net loss per share of common stock, basic and diluted

​

$

(0.73)

​

$

(0.90)

​

$

(1.52)

​

$

(1.66)

Weighted average common shares outstanding, basic and diluted

​

54,386,318

​

53,885,651

​

54,331,340

​

52,615,606

Comprehensive loss:

​

​

​

​

​

​

​

​

​

​

​

​

Net loss

​

$

(39,542)

​

$

(48,435)

​

$

(82,353)

​

$

(87,317)

Unrealized gain (loss) on marketable securities

​

​

(244)

​

​

26

​

​

(1,165)

​

​

31

Comprehensive loss

​

$

(39,786)

​

$

(48,409)

​

$

(83,518)

​

$

(87,286)

Fulfilling the Promise of Genetic Medicines for Central Nervous System DisordersCorporate PresentationAugust 2022NASDAQ GS: PASG

2 Forward-Looking StatementThis presentation includes “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectation about timing and execution of anticipated milestones, including our planned initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about our manufacturing plans and strategies; estimates regarding our cash forecasts; the expected impact of the COVID-19 pandemic on our operations; and the ability of our lead product candidates to treat their respective target CNS disorders. These forward-lookingstatements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,”“plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize PBGM01, PBFT02, PBKR03, PBML04, and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; and the other risks and uncertainties that are described in the Risk Factors section of our most recent filings with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this presentation. We do not undertake any obligation to publicly update any forward-looking statements except as required by law. By attending or receiving this presentation you acknowledge that you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made; you will be solely responsible for your own assessment of the market and our market position; and that you will conduct your own analysis and besolely responsible for forming your own view of the potential future performance of Passage Bio.

3 The Passage Bio AdvantageOur VisionTo fulfill the promise of genetic medicines by developing groundbreaking therapies that transform the lives of patients with CNS diseasesA corporate model designed for success PENN GTP PARTNERSHIPDEDICATED MANUFACTURING & ANALYTICSBROAD AND ROBUST PIPELINE

4 Penn GTP Partnership Leaders in AAV gene therapy World-class Gene Therapy ProgramFounded by renowned innovatorJames M. Wilson, M.D., Ph.D.Chief Scientific Advisor, Passage Bio Cutting-edge research and bioengineering Rigorous preclinical characterization to support product candidate selection Next-generation vector technologies and novel capsids Decades of proven gene therapy expertise 350+ full-time employees Strong connections with orphan disease community4

5 A Broad and Robust Pipeline with Global RightsTransformative therapies for CNS disorders across rare and large patient populations Program* Indication GeneDiscoveryPre-clinicalPhase 1/2PivotalPEDIATRIC PBGM01 † GM1 gangliosidosis GLB1 PBKR03 Krabbe disease GALC PBML04 Metachromatic leukodystrophy ARSAADULT PBFT02 Frontotemporal dementia GRN PBAL05 Amyotrophic lateral sclerosis C9orf72 Unnamed Huntington’s disease HTT & Undisclosed Unnamed Alzheimer’s disease Undisclosed Unnamed Temporal lobe epilepsy Undisclosed 17 total program license options•4 clinical-stage therapies•2 research-stage pipeline candidatesExploratory research programs in Alzheimer’s disease and TLE *8 additional CNS pipeline license options remain; 3 license options were previously exercised, and rights were subsequently returned to the University of Pennsylvania.† Program includes ongoing natural history study of infantile and juvenile GM1 gangliosidosis patients

6 A Diversified Portfolio StrategyCreating sustainable value across rare and large, monogenic and non-monogenic CNS indicationsCutting-edge R&D ADULT (RARE, MONOGENIC)ADULT (NON-MONOGENIC)PEDIATRIC (RARE, MONOGENIC)3 programs•GM1 Gangliosidosis•Krabbe Disease•Metachromatic Leukodystrophy3 programs•Frontotemporal Dementia•Amyotrophic Lateral Sclerosis•Huntington’s Disease2 target ID research programs•Temporal Lobe Epilepsy•Alzheimer’s Disease<5K patients 5–200K patients>200K patients

7 Passage Bio Pilot Manufacturing SuiteScale-up Capability Supporting R&D Pipeline and Future Development Plans UPenn GTPResearch Analytics and Vector Engineering Catalent Suite and Global Supply ChainDedicated cGMP Manufacturing Capacity and End-to-End Clinical Supply Chain Passage Bio CMC R&D Lab Analyticaland Process Development and Clinical Product Testing Passage Bio Manufacturing and CMC CapabilitiesEnsuring product supply from clinical development through commercialization Dedicated Capacity and End-to-End Supply ChainKey Strategic PartnershipsInternal State-of-the-Art CMC CapabilitiesGROWTH >>>FOUNDATION >>>>PARTNERSHIP>>>>>>INVESTMENT >>>>>>>>>

PBGM01GM1 Gangliosidosis

9 GM1 Gangliosidosis: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GLB1gene mutations characterized by destruction of neurons in the brain and spinal cord.Characterized by rapidly progressive neurological decline resulting in reduced muscle tone, progressive CNS dysfunction, deafness, blindness, rigidity and skeletal dysplasia. RARE AND UNDERSERVEDpopulations with incidence of up to ~1 per 100,000 live births worldwide.No disease-modifying therapies are presently approved.Source: NIH, CHOP, American Journal of Neuroradiology

10 PBGM01OUR APPROACHNext-generation, proprietary capsid delivers functional GLB1gene encoding β-gal to the brain and peripheral tissuesPRECLINICAL EVIDENCECompelling preclinical data in knock-out mouse model•Dose-related histological correction, improvements in neurological function, and survival•Meaningful transduction of both CNS and critical peripheral organsCLINICAL DEVELOPMENT•Ongoing global Phase 1/2 Imagine-1 trial focused on early and late infantile GM1•Well-tolerated, positive safety profile, demonstration of functional transgene expression, and meaningful gains in developmental milestones*Potential transformative therapy for rare, underserved disorder * Based on Cohort 1 interim results

11 Imagine-1: Global Phase 1/2 Trial with PBGM01Initial safety and biomarker data from Cohorts 2 & 3 expected in 2H 2022 Trial DesignPhase 1/2, multi-center, open-label, dose escalation and confirmatory study Route of AdministrationIntra-cisterna magna (ICM)VectorAAVhu68DurationTwo years, with rollover into a separate long-term follow-up studyPrimary Endpoints•Safety and tolerability•Efficacy (confirmatory cohort)Regulatory Clearances and Designations•Received multiple global regulatory clearances•Received Orphan Drug, Rare Pediatric Disease and Fast Track designations by FDA and Orphan designation by EC COHORT 4Early Infantilen = 2DOSE 2 (1.1e11 GC/g)*DOSE 1 (3.3e10 GC/g)* Expansion CohortEarly Infantilen = 6 Expansion CohortLate Infantilen = 6 COHORT 2Late Infantilen = 2 COHORT 3Early Infantilen = 2 COHORT 1Late Infantilen = 2 60 days between subject enrollment IDMC review Recruiting Dosing complete* Genome copies per gram of estimated brain weight

12 Key Takeaways from Interim Cohort 1 Data*Well tolerated, demonstration of functional transgene expression, and meaningful developmental improvement BIOMARKERSPost treatment CSF and serum β-gal activity for both patients above natural history study (NHS) patient values SAFETYPBGM01 was well tolerated and had a positive safety profile•No serious adverse events (SAEs)•No complications related to ICM injection•No evidence of DRG toxicity CLINICAL STATUSMeaningful improvement across developmental areas on both the Vineland and Bayley scales including regaining of lost milestones* Interim data aspresented at ASGCT 25thAnnual Meeting on May 18, 2022.

13 Interim Clinical Results: Patient 1Patient 1 demonstrated meaningful gains in developmental milestones, advancing from baseline to 12-months•Gross motor: progressed from taking a few steps without support toobserved jumping, balancing on 1 foot, and climbing stairs•Expressive language: progressed from usingno words with meaning tousing names to address siblings and creating simple sentencesSustained improvement documented in all developmental areas *The Vineland-II is caretaker-assessed. **Patient 1 was subsequently found to have unrelated visual impairment that could have impacted the Bayley-III. (Patient 1 recently began wearing glasses.) The Bayley-III is based on direct observation by a neurodevelopmental specialist. 81318 232833 381315171921232527 Developmental Age (months) Chronological Age (months)Vineland-II* (12 months) Normal Fine motor Gross motor Receptive language Expressive language Personal living skills Interpersonal relationships Play and leisure time Overall 1012 14 161820 22 24 261315171921232527 Developmental Age (months) Chronological Age (months)Bayley-III**(12 months) Normal Gross motor Fine motor Receptive language Expressive language Cognition Overall

14 Interim Clinical Results: Patient 2At month 4, Patient 2 was clinically observed to have regained previously lost developmental milestones•Gross motor: progressed from standing with support for 5 seconds to standing with support for 1-3 minutes and walking without support•Expressive language: progressed from using no words with meaning to using single words and waving goodbyeContinued improvement despite having severe developmental delay at baseline and prior history of regression 7911 13 151719 212331333537 Developmental Age (months) Chronological Age (months)Vineland-II* (6 months) Normal Fine motor Gross motor Receptive language Expressive language Personal living skills Interpersonal relationships Play and leisure time Overall 357911131531333537 Developmental Age (months) Chronological Age (months)Bayley-III**(6 months) Normal* Gross motor Fine motor Receptive language Expressive language Cognition Overall*The Vineland-II is caretaker-assessed.**The Bayley-III is based on direct observation by a neurodevelopmental specialist.

15 •CSF β-gal activity increased above levels seen in NHS following dosing and sustained at upper limit of NHS•Serum β-gal activity increased in both patients; however, over time, post-treatment β-gal activity for Patient 1 was above NHS patient values, while Patient 2 normalized to NHS patient values•CSF GM1 gangliosides increased in Patient 1 but subsequently stabilized; data for Patient 2 fluctuated•Whole blood GM1 gangliosides in Patients 1 and 2 increased slightly through ~50 days post-treatment, then trended downward slightly over timeCohort 1 Interim Biomarker Data * Apparent estimated concentration1. Baseline reflects the average of two samples collected within 48-hours of dosing. †Based on preliminary data from University of Pennsylvania’s ODC NHS study (NCT04041102).β-Gal=β-galactosidase; CSF=cerebrospinal fluid; NHS=natural history study,WB=whole bloodPatient 2 Patient 1 0501001502000100200300400nM*Time (Days)CSF GM1 Gangliosides 01 2340100200300400β - Gal Activity, CSF (nmol/mL/3hr) Time (Days)CSF β-gal NHS patient value range0.30-1.81 nmol/mL/3hr† 051015200100200300400β - Gal Activity, Serum (nmol/mL/3hr) Time (Days)Serum β-gal1 NHS patient value range2.25-10 nmol/mL/3hr† 0200400600 8001,0000100200300400nM*Time (Days)WB GM1 Gangliosides

PBKR03Krabbe Disease

17 Krabbe Disease: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GALCgene mutations characterized by demyelination of neurons in the brain and periphery.Disease progression is rapid and highly predictable including loss of acquired milestones, staring episodes, peripheral neuropathy, seizures, blindness and deafness. RARE AND UNDERSERVEDpopulations with incidence of up to ~2.6 per 100,000 live births worldwide.No disease-modifying therapies are presently approved.Source: NIH, CHOP, American Journal of Neuroradiology, Third Party Research

18 PBKR03OUR APPROACHNext-generation, proprietary capsid delivers functional GALCgene encoding galactosylceramidase (GALC) to the brain and peripheral tissuesPRECLINICAL EVIDENCEPBKR03-treated Krabbe dogs had improved central and peripheral myelination, reduced neuroinflammation and increased survival rates with full phenotypic recoveryCLINICAL DEVELOPMENTOngoing global Phase 1/2 GALax-C trial focused on early infantile KrabbePotential transformative therapy for rare, underserved disorder

19 COHORT 2> 4m to <9mn = 3 COHORT 3> 1m to <4mn = 3 COHORT 1> 4m to <9mn = 4+GALax-C Global Phase 1/2 Trial with PBKR03Initial safety and biomarker data from subset of Cohort 1 expected by end of 2022 Confirmatory Cohort > 1m to <4mn = 6 Confirmatory Cohort > 4m to <9mn = 6 COHORT 4> 1m to <4mn = 3DOSE 2 (5.0e11 GC/g)*DOSE 1 (1.5e11 GC/g)* 60 days between subject enrollment Trial DesignPhase 1/2, multi-center, open-label, dose escalation and confirmatory studyRoute of AdministrationIntra-cisterna magna (ICM)VectorAAVhu68Duration2 years; with additional 3 years of follow-up for safety and durability of effectPrimary Endpoints•Safety and tolerability•Efficacy (confirmatory cohort)Regulatory Clearances and Designations•Received multiple global regulatory clearances•Received Orphan Drug, Rare Pediatric Disease and Fast Track designations by FDA and Orphan designation by EC IDMC review Recruiting* Genome copies per gram of estimated brain weight+Cohort 1 was expanded from 3 to 4 subjects per study protocol following a reported serious adverse event for Patient 1

PBFT02Frontotemporal Dementia —GRN

21 FTD-GRN: A Devastating Adult DiseaseDEVASTATING FORM OF DEMENTIA caused by a GRNgene mutation resulting in progranulin (PGRN) deficiency. Approximately 5–10% of FTD is caused by a GRNmutation.Disease progression is rapid and degenerative including loss of speech, loss of expression, severe behavioral changes and immobility. RARE AND UNDERSERVEDpopulations with estimated U.S. prevalence of ~3,000 to 6,000 patients.No disease-modifying therapies are presently approved.

22 PBFT02OUR APPROACHProprietary construct delivers functional GRNgene encoding progranulin (PGRN) with potential therapeutic benefit of a one-time gene therapy approachPRECLINICAL EVIDENCECompelling preclinical evidence from NHP studies•Broad transduction across the brain, including high transduction of ependymal cells•Demonstrated increases in CSF PGRN concentrations to >50-fold normal human CSF PGRN concentrationsCLINICAL DEVELOPMENTOngoing global Phase 1/2 upliFT-D trial focused on early symptomatic FTD-GRNPotential transformative therapy for rare, underserved disorder

23 upliFT-D: Global Phase 1/2 Trial with PBFT02First patient dosing expected in mid-2022 OPTIONAL DOSE 3 DOSE 2 (1.1e11 GC/g)* DOSE 1 (3.3e10 GC/g)* 60 days between subject enrollment COHORT 1n = 3 COHORT 2n = 3 OPTIONAL COHORT 3n = 3 Trial DesignPhase 1/2, multicenter, open-label, dose escalation studyRoute of AdministrationIntra-cisterna magna (ICM)VectorAAV1Duration2 years; with additional 3 years of follow-up for safety and durability of effectPrimary Endpoints•Safety and tolerabilitySecondary Endpoints•Biomarkers, functional and clinical signs of disease progressionRegulatory Clearances and Designations•Received regulatory clearances from FDA, Health Canada and ANVISA (Brazil)•Received Orphan Drug and Fast Track designations by FDA and Orphan designation by EC IDMC review Recruiting* Genome copies per gram of estimated brain weight

PBML04Metachromatic Leukodystrophy

25 Metachromatic Leukodystrophy: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by ARSAgene mutations leading to accumulation of sulfatides in the brain and other areas of the body.Disease progression is rapid and highly predictable including muscle weakness, rigidity, gait disorder, developmental delaysRARE AND UNDERSERVEDpopulations with incidence of ~1per 100,000 live births worldwide.Limited disease-modifying therapies are presently approved.Source: NIH, CHOP,Kehrer et al. The natural course of gross motor deterioration in metachromatic leukodystrophy. Dev Med Child Neurol.2011.

26 PBML04OUR APPROACHNext-generation, proprietary AAVhu68 capsid delivers functional ARSAgene encoding arylsulfatase A (ARSA) to the brain and peripheral tissues via ICM injectionPRECLINICAL EVIDENCECompelling preclinical data in a knock-out mouse model•Dose-related lysosomal correction, improvements in neurological function, and increased survival•Meaningful transduction of both CNS and critical peripheral organsCLINICAL DEVELOPMENTIND clearance obtained from FDA in Q2 2022Potential transformative therapy for rare, underserved disorder

27 Summary•Novel ARSAknockout mouse model to assess effects on survival and clinical impairment•Dose-dependent improvements in survival and reduced clinical impairment observed•All mice in the AAV-treated groups survived while 5/10 mice in the untreated group died prematurely•Rate of clinical impairment reduced in AAV-treated mice, with highest dose group declining by only 2-3 points more than age-matched wildtype control mice PBML04 (ICV) dose-dependently reduced functional decline and increased survival in a novel MLD mouse modelComposite clinical scale in a novel ARSA-knockout line PBML04 –Metachromatic LeukodystrophyData provided by Penn GTPWTKO + Veh KO AA-oKO AA-ed KO AA-igh p < 0.005************5/1010/1010/1010/1010/10Survival 567891011121314151617181920 024 6 810 Sum of Deficit ScoresAge (Months) Untreated ARSA KO mouse* Max sum of deficit scores = 20*

Looking Ahead

29 TIMINGMILESTONEGM1Early 2022First patient dosed for Cohorts 2 and 3Feb 2022Late breaker presentation at WORLDSymposium2H 2022Initial safety and biomarker data for Cohorts 2 and 3FTD-GRNMid-2022First patient dosed in Phase 1/2 trial for PBFT02KrabbeEarly 2022First patient dosed in Phase 1/2 trial for PBKR03YE 2022Initial safety and biomarker data from subset of Cohort 1MLDMid-2022File IND for PBML041ManufacturingYE 2022Pilot plant operationalKey Milestones RESEARCH PIPELINE•Advance programs for ALS and Huntington’s Disease•Advance target ID research programs for Alzheimer’s Disease and TLE•8 additional CNS pipeline options remain through GTP partnershipBALANCE SHEET•Cash balance of $239 million as of 6/30/22*•Cash on hand to fund operations into Q2 2024* Cash, cash equivalents and marketable securities 1IND clearance obtained from FDA in Q2 2022.

30 30Genetic Medicines Company on a Mission to Transform the Lives of Patients with CNS Diseases A diversified rare and large portfolio strategy to ensure sustained value Robust collaboration with GTP, an unmatched leader in cutting-edge AAV gene therapy research Dedicated manufacturing and analytics to ensure capacity, flexibility and control

Thank Youpassagebio.com |NASDAQ GS: PASG

32 Demonstrated LeadershipLEADERSHIP TEAM Simona KingChief Financial OfficerMaria Törnsén Chief Commercial OfficerMark Forman, M.D., Ph.D.Chief Medical Officer Chip CaleInterim Chief Executive Officer &General CounselAlex Fotopoulos Chief Technology OfficerDeep experience in rare disease, CNS disorders and genetic medicines Desiree Luthman, D.D.S.SVP Global Regulatory AffairsBOARD OF DIRECTORSMaxine Gowen, Ph.D.InterimExecutive ChairwomanAthenaCountouriotis, M.D.Turning PointSaqib Islam, J.D.SpringWorksMichaelKamarck, Ph.D.Sanofi Biotech Advisory CouncilSandip KapadiaHarmony BiosciencesDerrell Porter, M.D.CellevolveBioTom Woiwode, Ph.D.Versant

33 Intra-Cisterna Magna (ICM) Administration•Directly deliver vector into the CSF via a single injection to reach both CNS and peripheral tissues*–Allows for broad CNS biodistribution–Lower doses compared to IV systemic delivery–Reduced impact of neutralizing antibodies •Administered under anesthesia using modern neuroimaging to allow for precise delivery•Currently being used in several clinical studies in both pediatric and adult populations Cisterna magna Source:Hinderer et. al,Human Gene Therapy. 2018 Jan; 29(1):15-24Utilized across three clinical-stage programs to directly target CNS

34 Pathway to Patient Identification and Trial Recruitment Screening / DiagnosisSponsoring ScreenPlus Pilot Program•GM1 included in the New York newborn screening pilot program led by Dr. Melissa Wasserstein Supporting Invitae Detect LSD and InformedDNA Programs•Free genetic testing and counseling offered to support earlier diagnosis and patient identification•Clinical trial information provided to clinicians and patients Patient and Caregiver ActivitiesImplementing caregiver and physician support and/or training programsDecreasing patient and site trial burden•Remote visits and video capture implemented for relevant endpoints•Clincierge offered for travel and accommodation support Patient Recruitment and Advocacy StrategiesMaintaining strong relationships with trial sites, study coordinators and investigatorsEstablishing clinical trial sites at centers of excellence globally Increasing clinical trial awareness •Partnering with patient advocacy groups, medical specialists and organizations