Issuer Free Writing Prospectus dated March 20, 2025
Filed Pursuant to Rule 433
Relating to Preliminary Prospectus Supplement dated March 20, 2025
Registration No. 333-276462
 Delivering on the Promise of Gene Therapy for Rare Inherited Retinal Diseases  Braydon,  RDH12 patient 
 
 Market Landscape & Commercial Opportunity  D i s e a s e O v e r v i e w  Most early-onset and severe form of all IRDs; severe vision loss during infancy or at an early age  Most common bestrophinopathy; presents in first two decades of life and causes progressive bilateral vision loss  U . S .  P r e v a l e n c e  ~200 patients  ~9,000 patients  P r o g n o s i s  Onset of profound vision loss typically during infancy, with 100% legally blind at an early age  Slow and progressive vision loss, with some progressing to legal blindness  T r e a t m e n t s  None  None (small proportion who develop CNV as a complication are treated with anti-VEGF therapy)  P i p e l i n e  None  None  H C P / P a t i e n t A d o p t i o n R a t e *  99% / 83%  82% / 61%  S u g g e s t e d  P r i c e  ~$500K per eye (all cases are bilateral)  ~$450K per eye (majority of cases are bilateral)  C o v e r a g e  R a t e  92%; Premium price to Luxturna®, with limited restrictions due to urgent need/small patient population  92%; Limited restrictions, with Luxturna as benchmark for price/coverage  L C A 5  B E S T 1  Luxturna® is a re gistere d trad emark of Spark Th erapeu tics, Inc.  *Based on ta rget p roduct pr ofile sugge sting improved vision .  2 BES T1, bestrophin 1; CNV, choroida l ne ova scularization; IRD, inhe rited retinal disease; LCA5 , Lebe r cong enital ama urosis 5; VEGF, vascu lar endothelial gro wth factor.  1. Stone et al. Op hth almology. 201 7;1 24:1314-133 1. 2. Triangle Insights Gr oup market r ese arch, conducted August 2 023. 
 
 One-year Results from a  Phase 1/2 Study of OPGx-LCA5 Gene Therapy for LCA5  Alan,  LCA5 patient  LCA5, Leber congenital amaurosis 5. 
 
 MLoMT utilizes a readily available VR headset with body trackers to navigate a virtual course  Household objects are presented at increasing illumination while the subject follows a path of red arrows  Subject identifies and “touches” obstacles while following the path  Establishes a threshold of functional vision that may be used to assess impact of disease and treatments  Enormous amount of data automatically collected  Relates well with clinical readouts (visual acuity, visual fields, and visual sensitivity)  MLoMT, Multi-luminance orientation and mobility test; VR, virtual reality.  4 Bennett J, et al. Transl Vis Sci Technol. 2023;12:28.  Easing the Answer to a Regulatory Need: Functional Vision Assessment with a Multi-Luminance Orientation and Mobility Test (MLoMT) 
 
 5  MLoMT Builds Upon the Success of MLMT®  Leverages VR to Provide Flexible Testing with Automatic Scoring  MLMT® and Multi-Luminance Mobility Test® are registered trademarks of Spark Therapeutics, Inc.  MLoMT, Multi-luminance orientation and Mobility Test; MLMT, Multi-Luminance Mobility Test; VR, virtual reality.  1. Bennett J, et al. Transl Vis Sci Technol. 2023;12:28; 2. Aleman et al. Clin Ophthalmol. 2021;15:939  Automatic randomization of dozens of configurations  Delivers test in a relatively short time (20 mins)  Equipment/space affordable  Easy to deploy and duplicate at multiple sites  No physical obstacles that could cause harm in a collision  Attractive to digital-savvy pediatric population  Quantitative information (timing, direction of gaze, acceleration, deceleration, collisions) captured automatically as digital data  Data obtained instantaneously and analyzed  objectively (no need for reading center)  MLMT Physical  Course  MLoMT Virtual Course 
 
 6  MLoMT: All Treated Subjects Identified More Objects Through 12 Months Compared to Baseline  L u m i n a n c e  CFB, change from baseline; FDA, Food and Drug Administration; MLoMT, Multi-Luminance orientation and Mobility Test.  S u b j e c t  0 1 - 03   O bj ects Id e nti f ie d  ( co un t )  S u b j e c t  0 1 - 01   O bj ects Id e nti f ie d  ( co un t )  O bj ects Id e nti f ie d  ( co un t )  S u b j e c t  0 1 - 04   Baseline Month 3  Month 6  Month 12  9 Normal-sighted subjects  9  8  7  6  5  4  3  2  8  7  6  5  4  3  2  1  0  9  8  7  6  5  4  3  2  1  1  0  -1.7 -1.2 -0.7 -0.3  Dimmest Brightest  L um in a nce ( lo g p h ot cd . m - 2 )  -1.7 -1.2 -0.7 -0.3  Dimmest Brightest  L um in a nce ( lo g p h ot cd . m - 2 )  0  -1.7 -1.2 -0.7 -0.3  Dimmest Brightest  L um in a nce ( lo g p h ot cd . m - 2 )  B L   M o n t h  3  M o n t h  6  M o n t h  1 2   B L   M o n t h  3  M o n t h  6  M o n t h  1 2   B L   M o n t h  3  M o n t h  6  M o n t h  1 2   - 1 . 7 (dimmest)  0  0 . 5  0  0  0  0  1 . 5  0  0  1 . 5  1 . 5  3  - 1 . 2  0  1 . 5  0  0  0  1  6  6  0  4 . 5  6 . 5  5 . 5  - 0 . 7  0  1  1 . 5  0 . 5  0  4 . 5  8 . 5  6  4 . 5  6  7  6 . 5  - 0 . 3 (brightest)  0  2  0  0 . 5  0  6  6 . 5  6 . 5  4 . 5  9  8  7  C o u n t o f O b j e c t s I d e n t i f i e d ( M a x = 9 )  Average CFB to 6 months at -0.7 luminance was 4 objects identified; This will be the possible primary endpoint in the FDA registrational study 
 
 7  Full-field Stimulus Testing: Demonstrated an Overall Improvement in Vision in Treated Eyes  Mean CFB: 0. 86 log improvement in study eyes vs 0. 16 in control eyes  Study eyes showed larger change from baseline, demonstrating improvement in vision  Pre-treatment interocular difference  Baseline: IOD average of 0. 3 log better sensitivity in control eyes  Post-treatment: IOD switched, with average of 0. 7 log better sensitivity in study eyes  BL, baseline; CFB, change from baseline; FST, full-field stimulus testing; IOD, interocular difference; SEM, standard error of the mean.  Study eye minus  control eye  Smaller symbols:  Individual values  Larger symbols:  Mean ± SEM  Smaller symbols:  Individual values  Larger symbols:  Mean ± SEM  Mean CFB in FST Mean IO D in F ST 
 
 8  FST: Retinal Sensitivity Gains Comparable to Adult Patients Treated with Luxturna  -4  -3  -2  -1  0  BL M1 M3 M6 M12  Mean sensitivity  [log phot-cd.s.m-2]  -4  -3  -2  -1  0  Mean sensitivity [log phot-cd.s.m-2]  -4  -3  -2  -1  0  BL M1 M3 M6 M12  Mean sensitivity  [log phot-cd.s.m-2]  -4  -3  -2  -1  0  Mean sensitivity  [log phot-cd.s.m-2]  -4  -3  -2  -1  0  BL M1 M3 M6 M12  Mean sensitivity [log phot-cd.s.m-2]  -4  -3  -2  -1  0  Mean sensitivity  [log phot-cd.s.m-2]  Su b j e c t 0 1 - 03 Su b j e c t 0 1 - 01 * Su b j e c t 0 1 - 04   D a r k - a d a p t e d r e d  L i g h t - a d a p t e d r e d  D a r k - a d a p t e d r e d  BL M1 M3 M6 M12 BL M1 M3 M6 M12 BL M1 M3 M6 M12  *Subject 01-01 was measured in light-adapted conditions; Remaining rod function was strong enough that it interfered with measuring cone function; to isolate and measure  cone responses, rods had to be desensitized by adapting the eye to white light  Note: Subject 01-03 and 01-04 are cone-mediated disease and Subject 01-01 is rod-mediated disease.  BL, baseline; FST, full-field stimulus testing; M, month.  D a r k - a d a p t e d b l u e  L i g h t - a d a p t e d b l u e  D a r k - a d a p t e d b l u e  Study eyes  Control eyes  Study eyes  Control eyes 
 
 FST: Improvement in Cone-mediated Vision in Treated Patients  Control Eye  Study Eye  S u b j e c t 0 1 - 03 D a r k - a d a p t e d  Proportion Seen [%]  Integrated Luminance [log phot-cd.s.m-2]  Control Eye  Study Eye  Proportion Seen [%]  Integrated Luminance [log phot-cd.s.m-2]  Control Eye  Study Eye  Proportion Seen [%]  Integrated Luminance [log phot-cd.s.m-2]  S u b j e c t 0 1 - 01 L i g h t - a d a p t e d  S u b j e c t 0 1 - 04   D a r k - a d a p t e d  FST data with red stimuli at Baseline and Month 12:  Study eyes: At 12 months, the proportion of stimuli seen shifted left toward lower luminance in all treated eyes, demonstrating better vision  Control eyes: Minimal to no change  BSL, baseline; FST, full-field stimulus testing’ M, month.  9 
 
 Pupillary Light Reflex: Results Supportive of Improved Cone-Mediated Vision Through 12 Months  Pupil responses were larger in study eyes  (green lines) compared to control eyes (black lines) and baseline (gray lines)  Treated eyes demonstrated a shift toward dimmer intensities compared to baseline  − Subjects 01-03 and 01-04 showed clear shift to the left confirming sensitivity gains  10 
 
 11  Visual Acuity: Overall Improvements in Treated Patients  CFB in Visual Acuity  Study eyes improved to 0.48 logMAR  (~5 lines on VA chart) at 6 months  Control eyes fluctuated within 0.18 logMAR, with no improvement  Mean IOD in Visual Acuity  Symmetry / no difference between eyes at baseline  Post-treatment IOD favored study eyes by average of 0.3 logMAR  CFB, change from baseline; IOD, intraocular difference; logMAR, logarithm of the minimum angle of resolution; VA, visual acuity.  Pre-treatment  interocular difference  No change  from baseline  Study eye minus control eye  Smaller symbols:  Individual values  Larger symbols:  Mean ± SEM 
 
 0.9  0.7  0.5  0.3  0.1  -0.1  -0.3  -0.5  -0.7  VA, CFB [logMAR]  0.9  0.7  0.5  0.3  0.1  -0.1  -0.3  -0.5  -0.7  VA, CFB [logMAR]  12  Visual Acuity: Improvements from Baseline Demonstrated in Two Patients Over 12 Months  0.9  0.7  0.5  0.3  0.1  -0.1  -0.3  -0.5  -0.7  VA, CFB [logMAR]  Control eyes Study eyes  S u b j e c t  0 1 - 03   S u b j e c t  0 1 - 01   S u b j e c t  0 1 - 04   Improvement  VA improvements over 12 months in patients 01 -03 and 01-04  Formed vision possible for the first time in the most severely affected patient (most advanced disease), 01-03  CFB, change from baseline; VA, visual acuity.  Improvement  Improvement  Baseline VA  Baseline VA  Baseline VA 
 
 Microperimetry Confirms Foveal Improvement: Subject 01-04 had Greater than 18-Fold Improvement in Macular Sensitivity  Study Eye  Baseline: Measurable only in scattered points of barely detectable sensitivity; Fixation unsteady, over area of ~10 degrees  1 Month: Improvement with detectable sensitivities in 19 points over area of ~15 degrees  Fixation stabilized and shifted toward foveal center, suggesting improved central vision  13 Note: Microperimetry only possible in one subject; Two subjects could not fixate.  Control Eye Small area of severely reduced sensitivities at  5 points with no change through  12 months 
 
 B a s e l i n e  D a y 9 0  4  4  4 8  12  10  14  18  8 10  14  10  14  Possible FDA Registrational Endpoint  7dB difference in the same 5 prespecified loci at two or more timepoints may be a standalone registrational endpoint  D i f f e r e n c e a t s e l e c t l o c i  FDA, Food & Drug Administration. 
 
 15  Uneventful Subretinal Injections and Unchanged Retinal Structure  Uneventful subretinal injections  Unilateral injection; 300 µl volume  Retinotomies (black dots) and resulting subretinal blebs (white dashes)  Blebs coalesced near foveal center (arrows)  No major changes in retina post-treatment  Retina reattached post-injection  OCT images through foveal center of treated eye at BL and 1 year after procedure  B L   12   M o n t h s  Functional retinal pigment epithelium  BL, baseline; OCT, optical coherence tomography. 
 
 No dose-limiting toxicities  No serious adverse events  Anticipated adverse events were mild and unrelated to treatment  − Mostly related to use of systemic steroid or with surgical procedure  All early AEs resolved within 30 days of the procedure  16  OPGx-LCA5 was Safe and Well-Tolerated Through One Year  AE, adverse event; LCA5, Leber congenital amaurosis 5. 
 
 Data from first three adult patients presented to IDSMC  Compelling data demonstrating efficacy  Signal akin to Luxturna  No toxicity  OPGx-LCA5 low dose = 1E10 vg/eye  Luxturna dose = 1.5E11 vg/eye, with some cases of post-approval  inflammation  Unanimous recommendation to push for a seamless 1/2/3 design with data-driven dose escalation  Independent Data Safety Monitoring Committee Recommendation  IDSMC, Independent Data Safety Monitoring Committee; LCA5, Leber congenital amaurosis 5.  17 
 
 “I initially agreed to participate in trial to help people, including a child in our own neighborhood who also has LCA5. I wanted this research to  advance understanding and treatments that could make a difference for him and so many others.  After surgery and recovery, I’ve found the trial and treatment has also helped me find more joy in the little things—now I'm able to see fireworks light up the sky and appreciate the food on my plate—and improve my ability to do daily tasks that those without retinal diseases may take for granted like spotting my Uber and using utensils. I look forward to the possibility of treating my other eye once the trial advances to Phase 2.”  - OPGx-LCA5 Clinical Trial Participant  A Patient’s Journey in the OPGx-LCA5 Trial: Helping Others and Life-changing Moments  “  “  LCA5, Leber congenital amaurosis 5.  18 
 
 OPGx-LCA5 Clinical Development Plan  Alan,  LCA5 patient  LCA5, Leber congenital amaurosis 5. 
 
 Primary endpoint: MLoMT ORT; Change from baseline in total number of objects recognized  − FDA pre-meeting feedback indicates openness to new primary endpoint  − Must provide to IND: Justification of endpoint relevance in LCA5, tools intended for assessing study outcomes, interview guide for planned cognitive interviews supporting MLoMT measure in LCA5, quantitative assessment of reliability and construct validity for interviews, scoring algorithm, and plans for handling missing data  Secondary endpoints:  Composite MLoMT measure incorporating orientation, total number  of objects recognized, along with luminance  FST is a key secondary endpoint  Pupillometry as additional secondary endpoint  FDA, Food and Drug Administration; FST, full-field stimulus testing; IND, Investigational New Drug; LCA5, Leber congenital amaurosis 5;  20 MLoMT, multi-luminance orientation and mobility testing; ORT, object recognition threshold.  OPGx-LCA5 Primary and Secondary Endpoints  F D A D i s c u s s i o n / A g r e e m e n t  FDA meeting: March 3, 2025  Final FDA minutes: April 3, 2025  Pre-Type D meeting comments:  FDA open to adaptive Phase 1/2/3  clinical trial design proposed  Primary control is change from  baseline assessments of  MLoMT (6-month run-in  compared to 6-month post-  treatment)  FDA open to potentially skipping  further dose escalation if 3  adolescent LCA5 subjects show response of MLoMT ORT of 3 or more 
 
 A Precedent for Future Trials  Quicker Time to Approval  2028 vs 2031  Rationale for Adaptive Design for OPGx-LCA5 Clinical Trial  Lower Clinical  Costs  5M vs 7M 30% reduction  21  LCA5, Leber congenital amaurosis 5. 
 
 FDA Approves Adaptive Phase 1/2 Trial Design and Streamlined CMC Package  BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; FDA, Food and Drug Administration;  22 LCA5, Leber congenital amaurosis; MLoMT, Multi-luminance orientation and Mobility Test; ORT, object recognition threshold.  3 Pediatric  Q1-Q2 2025  Dosing  3 Adult  Treated, 1.5 yr  Follow Up  Phase 1/2 Cohort 1 (Low Dose: 1E10 vg/eye)  +Day 30 Safety Eval  +3 ORT in  MLoMT at 6 Months in  3 Pediatric  Subjects  Convert to Pivotal in Pediatric Subjects (Low Dose: 1E10 vg/eye)  15 Pediatric Subjects  (Q4 2026 – Q3 2027 Dosing)  Achieved  Includes 6-month run-in to collect primary  endpoint data, allowing:  Establishment of an LCA5-specific MLoMT reference data set  Subjects to be used as their own controls  CMC: Requesting FDA to allow manufacture of only 1 batch  1 batch will provide enough vector to treat entire  U.S. LCA5 population  In Pre-Type D comments, FDA seems open to path to BLA without new batch, batch production may  only be for post-approval (commercial) use 
 
 2028  CMC  Patient  Enrollment  EOP2  Run-In  Pivotal Data  BLA  Enroll & Treat  D30 data  M3 data  M6 data  Prepare BD  EOP2 meeting  Protocol, site contracting  Protocol to FDA  IRB approval  Enroll 16 patients  Treat 16 patients  Prepare BLA  File BLA  5 pts enrolled  6M data on 5 pts  Submit revised protocol  IRB approval  M6 data  6M data all pts  2025  Q1 Q2 Q3  Full GMP Commitment  Q4  2026  Q1  Q2  Q3 Q4  Mfg & release  2027  Q1  Q2  Q3  Q4  2028  Q1  Q2  Submit MR  OPGx-LCA5 Timelines  Today  BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; EOP, end of phase; FDA, Food and Drug Administration; GMP, Good  Manufacturing Practices; IRB, Institutional Review Board.  23 
 
 OPGx-LCA5 Use of Funds  BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; CRO, contract research organization; FPI, first patient in;  24 IND, Investigational New Drug; LPI, last patient in.  $(000s)  CMC  Pre-/Non-clinical  Clinical  Total  2025  Q1  $0  $25  $244  $269  Q2  $0  $25  $244  $269  Q3  $600  $60  $244  $904  Q4  $1,000  $60  $244  $1,304  Total  $1,600  $170  $975  $2,745  2026  Q1  $2,600  $30  $368  $2,998  Q2  $2,600  $30  $368  $2,998  Q3  $1,600  $30  $368  $1,998  Q4  $100  $30  $368  $498  Total  $6,900  $120  $1,470  $8,490  2027  Q1  $125  $30  $352  $507  Q2  $125  $30  $352  $507  Q3  $125  $30  $352  $507  Q4  $125  $30  $352  $507  Total  $500  $120  $1,410  $2,030  2028  Q1  $125  $0  $309  $434  Q2  $125  $0  $309  $434  Q3  $125  $0  $309  $434  Q4  $125  $0  $309  $434  Total  $500  $0  $1,235  $1,735  2025-2028 Total  $9,500  $410  $5,090  $15,000  K e y P r o g r a m A s s u m p t i o n s  IND  Complete  Pivotal  FPI (treated): Nov 2026,  LPI July 2027  BLA  Q2 2028  CMC  Start analytical work Q3 2025, 1 commercial batch  Other  Nonclinical: Gene expression- and function-based potency assay development (in house + assay transfer and validation at a CRO) 
 
 OPGx-BEST1  Phase 1/2-Ready Gene Therapy for  BEST1-associated Disease  BEST1, bestrophin 1. 
 
 Traditional 5x3 dose escalation is partially skipped if evidence of full-fluid resolution within 6 months after treatment is seen in Cohort 1 (or Cohort 2)  At first occurrence of full fluid resolution in majority (2/3) of a dose cohort, converts to pivotal study extension with 1:1 randomization with crossover (low dose - 1E10 vg/eye)  Becomes adaptive Ph 1/2/3 approach like the proposed OPGx-LCA5 adaptive design  26  OPGx-BEST1 Strategy  ARB, autosomal recessive BEST1; BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy.  O P G x - B E S T 1 D U O - 1 0 0 1  Design: Phase 1b/2a, open-  label, non-randomized, single  ascending dose escalation,  safety and tolerability study of  OPGx-BEST1 in subjects with  BVMD or ARB (Basket approach  with traditional 3x3 design)  Initial dosing: ARB patients only  Up to 3 doses evaluated: 1.4 E9  vg/eye (cohort 1), 4.5 E9 vg/eye (cohort 2), and 2.25 E10 vg/eye (cohort 3); unilaterally injected 
 
 Pi vot a l  Design: 1:1 drug vs control (with crossover)  Subjects: 30, 6-month endpoint to file  Trial sites: 8 (TBD)  FPI: Q1 2027  LPI: Q1 2028  6-month data: Q2 2028  File BLA: Q3 2028  OPGx-BEST1 Clinical Development Assumptions  Ph a se 1 / 2  Subjects: 5 Pediatrics at low dose, no escalation  Trial sites: 3 (1 EU, 2 US)  FPI: Oct 2025  LPI: Jan 2026  27  BEST1, bestrophin 1; BLA, Biologics License Application; FPI, first patient in; LPI, last patient in. 
 
 OPGx-BEST1 DUO-1001 Clinical Design  Cohort 1: Low Dose  (1.4E9 vg/eye)  1st Dose 2025  1st Cohort will include Autosomal  Recessive BEST1 (ARB) subjects  OCT Shows Full Fluid Resolution within  6 Months  Cohort 3: High Dose  (2.25E10 vg/eye)  Cohort 2: Mid Dose (4.5E9 vg/eye)  1st Dose  Q3 2026  1st Dose  Q1 2026  28  Convert to pivotal Phase 3 extension of pediatric patients  (low dose – 1.4E9 vg/eye)  30 Subjects  BEST1, bestrophin 1; IDMC, Independent Data Monitoring Committee; OCT, optical coherence tomography. 
 
 OPGx-BEST1 Timelines  BEST1, bestrophin 1; BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; CRO, contract research organization; D, day; DP, drug product;  EOP, end of phase; FDA, Food and Drug Administration; FU, follow-up; GMP, Good Manufacturing Practices; IND, Investigational New Drug; IRB, Institutional Review  29 Board; M, month; P, patient. 
 
 OPGx-BEST1 Use of Funds  30  $(000s)  CMC  Pre-/Non-clinical  Clinical  Total  2025  Q1  $400  $36  $271  $790  Q2  $200  $36  $271  $590  Q3  $200  $36  $271  $1,590*  Q4  $200  $36  $271  $490  Total  $900  $144  $1,085  $3,129  2026  Q1  $3,000  $30  $476  $3,708  Q2  $2,200  $30  $476  $2,908  Q3  $3,600  $30  $476  $4,308  Q4  $100  $30  $476  $4,058**  Total  $8,900  $120  $1.903  $14,173  2027  Q1  $100  $30  $443  $693  Q2  $100  $30  $443  $693  Q3  $100  $30  $443  $693  Q4  $100  $30  $443  $693  Total  $400  $120  $2,293  $2,293  2028  Q1  $100  $0  $412  $647  Q2  $100  $0  $412  $647  Q3  $100  $0  $412  $2,147  Q4  $100  $0  $412  $647  Total  $400  $0  $1,649  $2,049  2025-2028 Total  $10,600  $384  $6,410  $21,644  K e y P r o g r a m A s s u m p t i o n s  IND  File Aug 2025  Phase 1  FPI: Oct 2025  LPI: Jan 2026  Pivotal  FPI: Q1 2027  LPI: Q1 2028  BLA  Q3 2028  CMC  Initiate full GMP lot after human POC  Other  Nonclinical: gene expression and function-based potency assay development  *includes $1,000 license milestone  **includes $3,250 license milestone  BEST1, bestrophin 1; BLA, Biologics License Application; CMC, chemistry, manufacturing, and controls; CRO, contract research organization; FPI, first patient in; GMP, Good Manufacturing Practices; IND, Investigational New Drug; LPI, last patient in; POC, proof of concept. 
 
 Timing and Use of Funds  OPGx-LCA5 and OPGx-BEST1  BEST1, bestrophin 1; LCA5, Leber congenital amaurosis 5. 
 
 BEST1, bestrophin 1; BLA, Biologics License Application; EOP, end of phase; G&A, general and administrative; GMP, Good Manufacturing Practices;  32 IND, Investigational New Drug; LCA5, Leber congenital amaurosis 5; M, month.  OPGx-LCA5 & OPGx-BEST1 Timing and Use of Funds  Cash runway  Cash runway to 2 approvals  $15M  $22M  $48M  Q1  G&A  $12M  $12M  Confidential. Do Not Distribute. | March 2025  $12M  $12M  In-line filter 
 
 Every  patient’ s eyes tell a story  Images of real patients with IRDs.  Confidential. Do Not Distribute. | March 2025 
 
 34  OPGx-LCA5 Phase 1/2 Study: Summary of Patient Demographics  BL, baseline; HM, hand motion; LCA5, Leber congenital amaurosis 5; OD, right eye; OS, left eye; SRI, subretinal injection.  Age  26  34  19  Sex  Male  Female  Female  Study eye  OS  OS  OD  Date of dosing  Aug 7, 2023  Sep 11, 2023  Nov 13, 2023  LCA5 variants; Allele 1/Allele 2  Gln279/Gln279  Arg255Gln/del.Exon1  Arg255/Arg255  Visual acuity  OD: HM  OS: HM  OD: 20/300  OS: 20/400  OD: 20/200  OS: 20/200  Refraction  OD: +3.00  OS: +3.00  OD: -1.50  OS: -2.50  OD: +2.25  OS: +2.00  Foveal  thickness (m)  BL   OD: 121  OS: NA  OD: 123.5  OS: 127.1  OD: 232.2  OS: 226.5  3M  OD: 136  OS: 133.5  OD: 127.1  OS: 119.3  OD: 170.8  OS: 205.1  S u b j e c t 0 1 - 03 S u b j e c t 0 1 - 01 S u b j e c t 0 1 - 04   Null or non-functional proteins, poor VA, and severe photoreceptor loss (thin retinas/foveas) 
 
 35  OPGx-LCA5 Phase 1/2 Study: Study Endpoints  AE, adverse event; BCVA, Best corrected visual acuity; DLT, dose-limiting toxicity; DMLMT, digital multi-luminance mobility test; FDT, frequency doubling technology; FST, full-field sensitivity testing; LA-TPLR, light-adapted transient pupillary light reflex; LCA5, Leber congenital amaurosis 5; MLCVA, mesopic, low-contrast visual acuity; mVFQ-25, Modified National Eye Institute Visual Functioning Questionnaire-25; OCT, optical coherence tomography; TPLR, transient pupillary light reflex; VR-O&M, virtual reality orientation and mobility.  Dark-adapted FST  BCVA  Oculomoter control and fixation stability  Dark-adapted TPLR  mVFQ-25  MLCVA  P r i m a r y : S a f e t y / To l e r a b i l i t y S e c o n d a r y : E f f i c a c y  E x p l o r a t o r y :  E f f i c a c y  Visual function/functional vision  Microperimetry  Kinetic perimetry  VR-O&M test (or DMLMT)  FDT perimetry  LA-TPLR  Additional methods as warranted  Immunogenicity  Neutralizing and binding antibodies to AAV8  Viral shedding  Antibodies to LCA5 protein  ELISpot assay for T cell responses  DLT events  Procedure-related AEs  Number/severity of AEs  OCT: Changes in total and outer retinal thickness 
 
 36  OPGx-LCA5 Phase 1/2 Study: Ocular Adverse Events  Subject Sign or Symptoms  Eye  Unexpected AE  Date of onset  Date of resolution  Surgery related?  Oral steroid- related?/Taper onset  Action taken  01-01  Eye pain  Study (OS)  No  Sep 11  Sep 12  Yes  No  Concomitant medication  Corneal abrasion  Study (OS)  Yes  Sep 18  Sep 18  No  No  Topical antibiotic and eye patching  01-03 Eye pain  Study (OS)  Yes  Aug 7  Aug 8  Yes  No  Concomitant medication  Subretinal  01-04 hyper reflexives  Study (OD)  Yes  Nov 13  Ongoing  Yes  No  Oral prednisone  LCA5, Leber congenital amaurosis 5; OD, right eye; OS, left eye. 
 

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