Precision therapeutics
for genetically defined
diseases

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

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

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

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

Breakthrough computer-aided
drug design
for transformative medicines

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

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

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

Transformative treatments for
people with obesity and other
cardiometabolic diseases

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

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

ADC specialist
for solid
cancers

Pioneering transformative new
biotherapeutics for patients
(NASDAQ: SYBX)

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

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

Targeting progranulin and
lysosomal biology to treat
neurodegenerative diseases

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

Novel therapeutics
for severe
respiratory diseases

Leading the
next frontier in
RNA therapeutics

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

Engineered
B cells
as medicines

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

Next generation ocular gene
therapies for vision restoration
and vision preservation

Discovering precision
covalent medicines for
immune diseases

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

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

Precision therapeutics for
genetically-defined
dry AMD

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

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

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

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

Reprogramming RNA processing
to address underlying
drivers of disease

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

Discovering new medicines
for severe
neurodegenerative diseases

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

Target-first, rational
molecular glue
drug discovery platform

New class of
regulated cell and
gene therapies

Utilizing the innate immune
system to treat cancer and
inflammatory diseases

Developing highly effective
and differentiated treatments
for cystic fibrosis

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

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

Harnessing the power of the epigenome to develop precision genomic therapeutics

Innovative treatments for
hematologic diseases
(NASDAQ: IRON)

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

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

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

Targeting regulators of
innate and adaptive immunity
to restore immune homeostasis

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

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

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

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

Novel therapeutics
targeting
diseases of aging

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

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

Developing treatments for
complex rare diseases
(NASDAQ: LRMR)

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

Restoring ABC transporter
function for patients with
serious genetic diseases

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

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

Bispecific antibodies
to improve the treatment
of serious diseases

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

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

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

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

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

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

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

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

Targeting RNA-modifying
proteins for novel
precision cancer therapies

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

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

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

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

Building
the future of
gene therapy

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)