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UCB Media Room: UCB strengthens its gene therapy activities


[12/11/2020 | 07:05]

** UCB strengthens its gene therapy activities with additional pipeline pro=
grams, capabilities and platforms

=C2=B7 Acquisition of Handl Therapeutics BV augments UCB=E2=80=99s existing=
early gene therapy pipeline with two research programs, a proprietary aden=
o-associated virus (AAV) capsid technology platform and capabilities=C2=A0
=C2=B7 New R&D collaboration with Lacerta Therapeutics provides access to a=
novel gene therapy program and proprietary AAV capsids
=C2=B7 Gene therapy investment strengthens UCB=E2=80=99s capabilities to di=
scover and develop differentiated solutions with unique outcomes for patien=

BRUSSELS, BELGIUM (12th November 2020, 07:00 CET) =E2=80=93 UCB today annou=
nced the acquisition of Handl Therapeutics BV, a rapidly growing and transf=
ormative gene therapy company based in Leuven, Belgium and a new collaborat=
ion with Lacerta Therapeutics, a Florida based clinical stage gene therapy =
company. The new acquisition and collaboration will together serve to rapid=
ly accelerate UCB=E2=80=99s ambition in gene therapy.

UCB has a vision to move from symptomatic treatments to disease modificatio=
n and eventually towards a cure. AAV-mediated gene therapy offers to delive=
r that potential and drive a fundamental change in how diseases are treated=
with the ability to remove or add disease-related proteins with a single t=
reatment. A vast array of diseases are amenable to gene therapy and UCB is =
embracing this modality to expand its capabilities and ultimately transform=
the lives of patients with severe diseases.

=E2=80=9CHandl Therapeutics BV was formed with the goal of harnessing today=
=E2=80=99s new frontiers in science and technology, and delivering transfor=
mative and advanced medicines to patients,=E2=80=9D said Professor Michael =
Linden, Founder and Chief Scientific Officer at Handl Therapeutics BV. =E2=
=80=9CKnowing that UCB shares this goal too gives me great confidence that =
our combined resources and expertise will provide the best possible chance =
of making this a reality and I am happy that we are joining the UCB family.=

Founded in 2019, Handl Therapeutics BV has a vision to deploy the power of =
disease modifying in vivo gene therapy to treat complex neurodegenerative d=
iseases through AAV capsid technology. Operating in a highly collaborative =
manner, Handl Therapeutics BV has built a strong international network to a=
ccess global capabilities and expertise. To this end, it combines state of =
the art technology platforms and scientific advances licenced from KU Leuve=
n (Belgium), Centre for Applied Medical Research (CIMA Universidad de Navar=
ra, Spain), University of Chile (Chile) and King=E2=80=99s College London (=
UK) to address unmet medical needs.=C2=A0

Florent Gros, Handl Therapeutics BV Founder and Chief Executive Officer sai=
d, =E2=80=9CUCB=E2=80=99s global footprint and scientific expertise in neur=
odegenerative diseases, coupled with our shared cultures of scientific adva=
ncement and commitment to patients, creates an exceptional environment in w=
hich we can accelerate the development of gene therapies and change patient=
s=E2=80=99 lives.=E2=80=9D

The Handl Therapeutics team will continue to be based in Leuven, Belgium, a=
nd will work very closely with UCB=E2=80=99s international research teams.=

In addition to the Handl Therapeutics BV acquisition, today=E2=80=99s annou=
ncement of a new collaboration with Lacerta Therapeutics underlines UCB=E2=
=80=99s strategic focus in gene therapy to fulfil its Patient Value Ambitio=
n. These transactions build upon the strategic acquisition of Element Genom=
ics, Inc. (acquired in 2018) that strengthened UCB=E2=80=99s genomics and e=
pigenomics research platforms aiding the identification of novel drug targe=

Founded in 2017, and a spin-off from the University of Florida, Lacerta The=
rapeutics=E2=80=99 mission is to make AAV-based therapies available for all=
patients with rare and serious neurological disorders. The research collab=
oration and licensing agreement with UCB will focus on a central nervous sy=
stem (CNS) disease with a high unmet need. Lacerta Therapeutics will lead r=
esearch, preclinical activities and the early manufacturing process develop=
ment, while UCB will complete IND-enabling studies, manufacturing and clini=
cal development. This new collaboration will allow UCB to access Lacerta Th=
erapeutics=E2=80=99 expertise in AAV-based CNS targeted gene therapies, for=
tifying UCB=E2=80=99s ability to produce effective treatments for neurodege=
nerative diseases.=C2=A0

=E2=80=9CAt Lacerta Therapeutics, we have dedicated our careers to the deve=
lopment of AAV gene therapy platforms and it remains our mission to advance=
these platforms and develop novel therapeutics for patients with neurodege=
nerative disorders,=E2=80=9D said Dr. Edgardo Rodr=CE=AFguez-Lebr=C3=B2n, P=
resident and Chairman of the Board at Lacerta. =E2=80=9CWe are looking forw=
ard to entering a new era, working in partnership with UCB. Our hope is tha=
t our combined expertise will lead to significant advances in identifying t=
reatments for orphan CNS diseases.=E2=80=9D

Dhavalkumar Patel, UCB=E2=80=99s Chief Scientific Officer said, =E2=80=9CUC=
B=E2=80=99s ambition for patients relies on our ability to innovate and del=
iver highly differentiated medicines. The acquisition of Handl Therapeutics=
BV and the new partnership with Lacerta Therapeutics offers us the potenti=
al to drive a fundamental change in how diseases are treated, by moving us =
from treating symptoms to disease modification and eventually towards a cur=
e.=E2=80=9D He added, =E2=80=9CWe are delighted to be able to welcome a ric=
h diversity of talent and expertise from both Handl Therapeutics BV and Lac=
erta Therapeutics. With their deep and wide-ranging knowledge, novel gene t=
herapy platforms and drive for innovation, I am confident that together we =
will transform the lives of people living with severe neurodegenerative dis=

This acquisition and new collaboration do not impact UCB=E2=80=99s financia=
l outlook for 2020.

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company =
focused on the discovery and development of innovative medicines and soluti=
ons to transform the lives of people living with severe diseases of the imm=
une system or of the central nervous system. With more than 7,600 people in=
approximately 40 countries, the company generated revenue of =E2=82=AC 4.9=
billion in 2019. UCB is listed on Euronext Brussels (symbol: UCB). Follow =
us on Twitter: @UCB_news.

About Handl Therapeutics BV
The vision of Handl Therapeutics is to deploy the power of disease modifyin=
g in vivo gene therapy to treat complex neurodegenerative diseases to begin=
addressing significant unmet medical needs in healthcare. The innovative b=
usiness model is based on a highly integrative approach that is built on ti=
ght links with an international network of academic partners. Handl Therape=
utics goal is to build a streamlined business that is designed to deliver t=
ransformative advanced medicines to the market.

About Lacerta Therapeutics
Lacerta Therapeutics Inc (www.lacertatx.com) is a clinical-stage gene thera=
py company that is leveraging its proprietary adeno-associated virus (AAV) =
vector technology and manufacturing platforms to develop treatments for cen=
tral nervous system and lysosomal storage diseases. Currently, Lacerta is f=
ocused on gene therapy solutions for Sanfilippo Syndrome Type B, Friedreich=
=E2=80=99s ataxia, Spinocerebellar ataxia, Pompe disease, and Alzheimer=E2=

Forward looking statement =E2=80=93 UCB
This press release contains forward-looking statements based on current pla=
ns, estimates and beliefs of management. All statements, other than stateme=
nts of historical fact, are statements that could be deemed forward-looking=
statements, including estimates of revenues, operating margins, capital ex=
penditures, cash, other financial information, expected legal, political, r=
egulatory or clinical results and other such estimates and results. By thei=
r nature, such forward-looking statements are not guarantees of future perf=
ormance and are subject to risks, uncertainties and assumptions which could=
cause actual results to differ materially from those that may be implied b=
y such forward-looking statements contained in this press release. Importan=
t factors that could result in such differences include: changes in general=
economic, business and competitive conditions, the inability to obtain nec=
essary regulatory approvals or to obtain them on acceptable terms, costs as=
sociated with research and development, changes in the prospects for produc=
ts in the pipeline or under development by UCB, effects of future judicial =
decisions or governmental investigations, product liability claims, challen=
ges to patent protection for products or product candidates, changes in law=
s or regulations, exchange rate fluctuations, changes or uncertainties in t=
ax laws or the administration of such laws and hiring and retention of its =
UCB is providing this information as of the date of this press release and =
expressly disclaims any duty to update any information contained in this pr=
ess release, either to confirm the actual results or to report a change in =
its expectations. There is no guarantee that new product candidates in the =
pipeline will progress to product approval or that new indications for exis=
ting products will be developed and approved. Products or potential product=
s which are the subject of partnerships, joint ventures or licensing collab=
orations may be subject to differences between the partners. Also, UCB or o=
thers could discover safety, side effects or manufacturing problems with it=
s products after they are marketed. Moreover, sales may be impacted by inte=
rnational and domestic trends toward managed care and health care cost cont=
ainment and the reimbursement policies imposed by third-party payers as wel=
l as legislation affecting biopharmaceutical pricing and reimbursement.

Contact information

Corporate Communications - UCB
Scott Fleming, E: scott.fleming@ucb.com, T: +7702 777 378
Laurent Schots, E: Laurent.schots@ucb.com, T: +32 (0)2 559 92 64

Investor Relations - UCB
Antje Witte, E: antje.witte@ucb.com T: +32 (0)2 559 94 14

Contact information =E2=80=93 Lacerta Therapeutics
Christina Sarabia, E: christinas@lacertatx.com, T: (1) 386-588-3285

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