Modalis Therapeutics Reports Operational Highlights andFiscal Year End 2023 Financial Results
14-Feb-2024 TOKYO & Waltham, Mass.- Modalis Therapeutics Corporation ("Modalis" 4883.T TSE), apioneer in developing cutting-edge solutions for rare genetic diseases through its proprietary
CRISPR-GNDM® epigenetic gene modification technology, today announced its financial results forthe fiscal year ended December 31, 2023, alongside recent operational milestones.
" While challenges are prevalent in the industry, we take immense gratification in witnessing theadvancement of MDL-101, navigating a clear-cut trajectory toward its Investigational New Drug(IND) status. The imminent prospect of its introduction into clinical trials as the inaugural CRISPR-based
epigenome-editing therapeutic raises enthusiasm within our ranks,” said Haru Morita, Chief Executive
Officer of Modalis. Morita further elaborated, "Our enthusiasm is compounded by the strategic
collaboration we forged with JCR Pharma (JCR) in December, which is focused on central nervous
system disorders. JCR's JBC technology, which enables genetic cargos to traverse the blood-brain
barrier (BBB), aligns seamlessly with our CRISPR-GNDM® platform. We believe the synergy between
these technologies will catalyze paradigm-shifting gene therapies, enabling us to precisely deliver
GNDM to discrete neuronal targets.”
Recent Preclinical and Business Highlightsl

MDL-101 is advancing to INDØØl

MDL-207 targeting Dravet Syndrome has been added to the pipelineØØl

Our innovative technology for epigenetic gene modulation is being explored as a potential long-term treatmentof this serious condition by increasing the expression of the SCN1a gene transcripts from surviving alleles.
Animal proof of concept has been established in a murine Dravet disease model.

Research collaboration is established with JCR PharmaØlReceived encouraging responses from FDA to our PreIND submission, elucidating a clear path to IND filing. Our
GLP-tox plans were aligned with FDA guidance and studies have been initiated.
Process development has been completed to enable GMP manufacturing.

We have initiated a research collaboration with JCR Pharma to develop a novel therapeutic for an undisclosed
CNS target utilizing JCR’s JBC-AAV technology and Modalis’ CRISPR-GNDM technology.

Three key patents have been granted or issuedØØProgress continues to be made with patents for elemental technologies being approved in various countries.
1. miniCas9 patent issued in Japan
2. PAM-flex SpCas9 patent granted in China
3. miniVR (small transcription activator) patent issued in Russia
Modalis also maintains its right and license to fundamental CRISPR patents, which Editas has recently licensed
to a third party for treatment for beta-thalassemia

Fiscal Year 2023 Financial Results:•Cash Position: Cash and deposits as of December 31, 2023, was ¥1,883 million, compared to¥2,933 million as of December 31, 2022, a decrease of ¥1,050 million. The decrease in Cashand deposits was primarily due to R&D Expenses and G&A Expenses.

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Revenues: No revenue for the full year ended December 31, 2023.
Research & Development (R&D) Expenses: R&D expenses were ¥2,103 million for full year
ended December 31, 2023, compared to ¥1,862 million for December 31, 2022, an increaseof ¥241 million. Increases in R&D expenses were primarily due to increases in development
costs associated with the advancement of the Company’s proprietary GNDM platform,
product candidates and yen depreciation against US dollar.
General & Administrative (G&A) Expenses: G&A expenses were ¥268 million for the fullyear ended December 31, 2023, compared to ¥242 million for December 31, 2022, anincrease of ¥26 million. Increases in G&A expenses were primarily due to increased

personnel costs.
Net Loss: Net Loss was ¥2,352 million for the full year ended December 31, 2023, comparedto ¥1,996 million for December 31, 2022.
Please refer to 4Q Consolidated Financial Results disclosed according to Japanese accounting
rules in the English version below.

About Modalis:
Modalis Therapeutics is developing precision genetic medicines through epigenetic gene
modulation. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPRGNDM technology which enables the locus-specific modulation of gene expression or histone
modification without the need for double-stranded DNA cleavage, gene editing or base editing.
Modalis focuses primarily on genetic disorders caused by loss of gene regulation – resulting in excess
or insufficient protein production – which includes more than 660 genes that are currently
estimated to cause human disease due to haploinsufficiency. Headquartered in Tokyo withlaboratories and facilities in Waltham, Massachusetts. For additional information,
visit www.modalistx.com.

Forward-Looking Statements:
This document has been prepared by Modalis Therapeutics corporation and Modalis Therapeutics
Inc. (the “Companies”) solely for information purpose only. This document does not constitute orform part of and should not be construed as, an offer to sell or issue or the solicitation of an offer tobuy or acquire securities of the Companies in Japan, the United States or any other jurisdictions. The
information contained herein is based on current economic, regulatory, market trends and otherconditions. The Companies make no representation or guarantee with respect to the credibility,accuracy or completeness of the information herein. The information contained herein may change
without prior notice. You may not publish or use this document and the contents thereof for any
other purpose without a prior written consent of the Companies. Furthermore, the information on
future business results are forward-looking statements. Forward-looking statements include but notlimited to expressions such as "believe", “expect", "plan", "strategic", "expect", "anticipate","predict" and "possibility", as well as other similar expressions to explain future business activities,achievements, events and future conditions. Forward-looking statements are predictions about thefuture that reflect management's judgment based on currently available information. As such, theseforward-looking statements are subject to various risks and uncertainties that could cause actual
results to differ materially from those expressed in or suggested by the forward-looking statements.
Therefore, you may not rely entirely on forward-looking statements. The Companies do not assume
any obligation to change or correct any forward-looking statements in light of new information,
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Contacts

Modalis Therapeutics
Sawako Nakamura
media@modalistx.com

Consolidated Financial Results
for the Fiscal Year Ended December 31, 2023
[Japanese GAAP]
Feb 14, 2024
Company name: Modalis Therapeutics Corporation
Stock exchange listing: Tokyo Stock Exchange
Code number: 4883
URL: https://www.modalistx.com/jp/
Representative: Haruhiko Morita, President and Representative Director
Contact: Naoki Kobayashi, CFO and Executive Officer
Phone: +81-3-6822-4584
Scheduled date of filing quarterly securities report: March26, 2024
Scheduled date of commencing dividend payments: Availability of supplementary briefing material on quarterly financial results: Available
Schedule of quarterly financial results briefing session: - Scheduled (for securities analysts and institutional investors)(Amounts of less than one million yen are rounded down.)
1. Consolidated Financial Results for the Fiscal Year Ended December 31, 2023 (January 1, 2023 to December31, 2023)(1) Consolidated Operating Results(% indicates changes from the previous corresponding period.)
Profit attributable toOperating revenue
Operating income
Ordinary income
owners of parent
Fiscal Year endedMillion yen%
Million yen%Million yen%
Million yen%
December 31, 2023
(100)
(2.370)(2.351)(2.391)
December 31 202240
(2,063)(1,995)(2,702)(Note) Comprehensive income: Fiscal Year ended December 31, 2023:: ¥ (2,400) million [- %]Fiscal Year ended December 31, 2022:: ¥ (2,691) million [- %]Basic earnings
Diluted earnings
Ordinary income toOperating income to
Return of equity
per shareper share
total assets
operating revenue%%%
Fiscal Year endedYenYen(111.9)(91.2)
December 31, 2023
(77.24)(63.8)(43.4)
December 31 2022
(92.85)(Notes)
The amount shown as “business revenue” in the consolidated statements of income has been shown as ”Operatingrevenue” in this document.
Diluted net income per share is not shown in the above table, because net income per share was negative although there
are residual shares.
(2) Consolidated Financial Position
Total assets

Net assets

Million yen

Net assets
per share

Million yen%

Yen

1,380
2,941

66.8
93.4

40.59
99.53

Cash flows fromoperating activities
Million yen

Cash flows frominvesting activities
Million yen

Cash flows fromfinancing activities
Million yen

Cash and cash equivalents
at the end of period(2,254)(1,895)

(39)
(185)

1,216
63

1,883
2,933

As of December 31, 2023
2,025
As of December 31, 2022
3,129
(Reference) Equity: As of December 31, 2023: ¥1,353 million
As of December 31, 2022: ¥2,922 million(3) Cash flow

As of December 31, 2023
As of December 31, 2022

Capital adequacy
ratio

Million yen