Building
the future of
gene therapy

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

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

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

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

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

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

Developing treatments for
complex rare diseases
(NASDAQ: LRMR)

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

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

Novel therapeutics
for severe
respiratory diseases

New class of
regulated cell and
gene therapies

Targeting RNA-modifying
proteins for novel
precision cancer therapies

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

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

Engineered
B cells
as medicines

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

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

Disease modifying therapies
for patients with rare
cardiopulmonary disease

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

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

Breakthrough computer-aided
drug design
for transformative medicines

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

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

Targeting progranulin and
lysosomal biology to treat
neurodegenerative diseases

Discovering new medicines
for severe
neurodegenerative diseases

Reprogramming RNA processing
to address underlying
drivers of disease

Developing medicines
that target inflammation
at its core

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

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

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

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

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

Next generation ocular gene
therapies for vision restoration
and vision preservation

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

Utilizing the innate immune
system to treat cancer and
inflammatory diseases

Leading the
next frontier in
RNA therapeutics

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

Applying new insights in
hepcidin biology to
treat hematologic diseases

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

Patient-directed oncology
therapies across immune and
cancer cell pathways

Precision therapeutics for
genetically-defined dry AMD
(NASDAQ: GMTX) Jan 2021

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

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

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

Harnessing the immune system
to defeat cancer with
tumor-selective immunotherapies

Bispecific antibodies
to improve the treatment
of serious diseases

Harnessing the power of microglia
to transform treatments for
neurodegenerative diseases

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

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

Targeting regulators of
innate and adaptive immunity
to restore immune homeostasis

Rapidly developing new
cancer treatments for
people of all ages

Precision therapeutics
for genetically defined
diseases

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

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

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

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

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

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

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

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

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

Novel therapeutics
targeting
diseases of aging

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

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

Designing Synthetic Biotic
living medicines to treat disease
(NASDAQ: SYBX)

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