Precision gene editing to
cure severe diseases –
IPO May 2016 (NASDAQ: NTLA)

Precision therapeutics for
genetically-defined
dry AMD

First-in-class RNA therapies
for severe genetic diseases
IPO Jun 2018 (NASDAQ: TBIO)

Broadening the reach and
impact of precision medicine
to more people with cancer

Developing treatments for
complex rare diseases
(NASDAQ: LRMR)

Innovative treatments for
hematologic diseases
(NASDAQ: IRON)

Translating epigenetic modulation of oncogenic pathways into first-in-class small molecule therapeutics

Enhancing synaptic integrity for
treatment of neurologic dis-
orders; sold to Alkermes Nov 2019

Reprogramming RNA processing
to address underlying
drivers of disease

Advancing medicines for
allergy and inflammation
IPO Sep 2022 (NASDAQ: THRD)

Rapidly developing new cancer
treatments for people of all ages
– IPO Jun 2021 (NASDAQ: DAWN)

Therapies for severe
cardiovascular diseases –
IPO Apr 2000 (SWX: ATLN)

Harnessing the immune system to
defeat cancer with tumor-selective
immunotherapies (NASDAQ: XLO)

Bispecific antibodies
to improve the treatment
of serious diseases

Targeting regulators of
innate and adaptive immunity
to restore immune homeostasis

Transformative treatments for
people with obesity and other
cardiometabolic diseases

Novel antibodies for immuno-
oncology – Sold to Novartis
(NYSE: NVS) Feb 2014

Breakthrough computer-aided
drug design
for transformative medicines

Preventing axonal degeneration in
patients with neurological diseases
Sold to Lilly (NYSE: LLY) Oct 2020

Genetic analysis to find novel
drug targets – Sold to Amgen
(NASDAQ: AMGN) Dec 2012

Restoring ABC transporter
function for patients with
serious genetic diseases

Precision therapeutics
for genetically defined
diseases

Pioneering targeted therapies
for serious muscle diseases –
IPO Sep 2020 (NASDAQ: DYN)

New class of
regulated cell and
gene therapies

Scalable, re-dosable, non-viral
gene therapy for genetic diseases
IPO Jun 2020 (NASDAQ: GBIO)

Novel bicyclic peptides (bicycles) and
bicycle drug conjugates for cancer
IPO May 2019 (NASDAQ: BCYC)

Novel therapies for severe, highly
drug-resistant bacterial infections
IPO Nov 2017 (NASDAQ: SPRO)

Migraine prevention therapy –
Sold to Eli Lilly (NYSE: LLY)
Dec 2013

Engineered
B cells
as medicines

Target-first, rational
molecular glue
drug discovery platform

Treating cancer’s toughest
tumors by harnessing the power
of precisely delivered radiation

Novel antibodies built to break
through the tumor microenvironment
IPO Apr 2018 (NASDAQ: SURF)

Leading the
next frontier in
RNA therapeutics

Anti-viral therapies –
Sold to AstraZeneca
(NYSE: AZN) Feb 2007

Novel biologic therapeutics for
autoimmune diseases – Sold to
Celgene (NASDAQ: CELG) Jan 2017

ADC specialist
for solid
cancers

Human antibody therapies for
oncology – Sold to Daiichi
Sankyo (TYO: 4568) May 2008

Targeting progranulin and
lysosomal biology to treat
neurodegenerative diseases

Targeting the DNA damage
response pathway to prevent and
halt expansion repeat disorders

Next-generation oncolytic immuno-
therapies for cancer treatment
– IPO Jul 2018 (NASDAQ: REPL)

Developing highly effective
and differentiated treatments
for cystic fibrosis

Restoring metabolic balance to treat
NASH and serious metabolic diseases
– IPO Jun 2019 (NADSAQ: AKRO)

Proprietary pharmaceuticals for
neonatal care and pediatrics –
Sold to Mallinckrodt Aug 2017

Mouse platforms for the generation
of fully human antibodies –
Sold to Harbour BioMed Dec 2016

Cell and gene therapies
for rare diseases
IPO Jun 2018 (NASDAQ: AVRO)

Development of complex
biopharma products – IPO
Jun 2004 (NASDAQ: MNTA)

Novel medicines for auto-
immune diseases – Sold to
BMS (NYSE:BMY) Mar 2016

Novel anesthetics –
Sold to The Medicines Company
(NASDAQ: MDCO) Jan 2015

Novel bio-therapies –
Sold to Bristol-Myers Squibb
(NYSE: BMY) Oct 2007

Advancing novel therapies for people
with rare cardiopulmonary disease
– IPO Jun 2021 (NASDAQ: AVTE)

Novel therapeutics using proprietary
computational chemistry –
Sold to Allergan (NYSE: AGN) Sep 2016

Utilizing the innate immune
system to treat cancer and
inflammatory diseases

Novel therapeutics
targeting
diseases of aging

Pioneering transformative new
biotherapeutics for patients
(NASDAQ: SYBX)

Cancer therapeutics and
diagnostics – Sold to Amgen
(NASDAQ: AMGN) Jan 2012

Genomics-based cancer
drug discovery – IPO
Apr 2000 (NASDAQ: EXEL)

World-class antibody
discovery platform – IPO
Mar 1999 (XETRA: MOR.DE)

Advancing targeted protein
degradation to target previously
“undruggable” disease pathways

Discovering precision
covalent medicines for
immune diseases

Unlocking nature’s regulatory
mechanisms to fuel the
discovery of new medicines

Harnessing the power of microglia to
transform treatments for neuro-
degenerative diseases (NASDAQ: VIGL)

Harnessing the power of the epigenome to develop precision genomic therapeutics

Discovering new medicines
for severe
neurodegenerative diseases

Delivering medicines for rare,
autoimmune, and inflammatory diseases
IPO July 2011 (NASDAQ: HZNP)

Covalent irreversible
inhibitors – Sold to Celgene
(NASDAQ: CELG) Jan 2012

Patient-directed oncology therapies
across immune and cancer cell pathways
IPO Mar 2021 (NASDAQ: IKNA)

Novel antibiotics –
Sold to AstraZeneca
(NYSE: AZN) Mar 2010

Novel therapeutics
for severe
respiratory diseases

Breakthrough therapies for neuro-
logical & psychiatric conditions
– Sold to Novartis Dec 2020

Building
the future of
gene therapy

RNAi-based drugs for
severe diseases – IPO
Jun 2004 (NASDAQ: ALNY)

Transforming stem cell transplant
therapy for auto-immune and blood-
based diseases (NASDAQ: MGTA)

Novel fibrosis therapeutic –
Sold to Biogen Idec
(NASDAQ: BIIB) Feb 2012

Novel vaccine platform –
Sold to Johnson & Johnson
(NYSE: JNJ) Oct 2010

Targeting RNA-modifying
proteins for novel
precision cancer therapies

Novel cancer drugs using
combinatorial chemistry –
IPO Oct 1996 (NASDAQ: ARQL)

Next generation ocular gene
therapies for vision restoration
and vision preservation