2020 Boosting translational medicine in Academia - Spark-BIH
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2020
Boosting
translational
medicine in
Academia
BIH
© SPARK-BIH | Berliner Institut für Gesundheitsforschung, November 2020
SPARK-BIH
c/o Berlin Institute of Health
Anna-Louisa-Karsch-Straße 2
10178 Berlin
spark@bihealth.de
www.spark-bih.de
SPARK program in Berlin.................................................................................................................................... 6 SPARK-BIH in Numbers – SPARK projects from 2015 – 2019............................................................................ 13 SPARK-BIH Projects – Our newly funded & mentored SPARK Teams of 2019........................................ 19 SPARK-BIH Projects – Our SPARK Teams that started funding & mentoring in 2015 - 2018 ............ 29 SPARK-BIH Projects – Mentored-only SPARK Teams ........................................................................................ 63 SPARK-BIH Projects – Our newly funded & mentored I4H Teams of 2019 ................................................... 67 SPARK-BIH Project Glossar................................................................................................................................. 76 SPARK-BIH Photo Copyrights.............................................................................................................................. 77 SPARK Team ............................................................................................................................................................ 78
SPARK program in Berlin
The SPARK program in Berlin was universities world-wide. The SPARK embedded within BIH Innovations, the
founded in 2015 by Prof. Dr. Craig Garner network serves as a resource for joint technology transfer of BIH and
and Prof. Dr. Ulrich Dirnagl with the knowledge, advisors and examples Charité. Thereby, SPARK in Berlin has
support of the Stiftung Charité. The of best practice. An annual meeting grown from supporting about 4 projects
goal was and is to support academic brings together the leadership of all per year with 50.000€ each to a program
inventors in translating their research SPARK programs to discuss global that currently funds and mentors almost
findings into therapies, products and health challenges, strategies to improve 30 project teams within the validation
services, such as novel therapeutics translation and how to augment fund. Employees from Charité, the Max
(small molecules, biologics, cellular the SPARK educational program. Delbrück Centre for Molecular Medicine
therapies), medical devices and An international workshop on Bio and the BIH can apply with translational
diagnostics for unmet medical needs. Innovation and Entrepreneurship brings projects from all medical areas.
together SPARK participants from all
The program in Berlin was modeled around the globe. Projects are selected for funding by
after the successful SPARK program the validation fund in an annual call
at Stanford University which Prof. In 2018 the Berlin Institute of Health for proposals with a panel of external
Dr. Garner was a member of. Since (BIH) decided to perpetuate the program experts. Selection criteria include the
its founding in 2006 SPARK has grown and to include SPARK-BIH as part of hight of innovation, size of the unmet
to a global network with programs their mission to support translational medical need, competitive advantage
implemented at more than 50 medicine. SPARK-BIH was successfully over current gold standards, data quality
4SPARK Team
and likelihood of translational success. SPARK educates faculty, students and and develop initial prototypes of their
Two funding tracks have been fellows on topics relevant for translation solutions. These teams are augmented
implemented in the validation fund: and business development via their by the addition of team-members from
Earlier projects are supported with up to educational series including forums and other disciplines such as business
50.000€ for one year in track 1 whereas workshops. and design to create truly holistic
later and more mature projects are solutions. In future it is planned that
supported for two years with a budget As of 2019, the Inventors for Health the most promising ideas can apply to
of more than 50.000€ as part of track Program has been implemented at be further developed by SPARK-BIH and
2. Funding is strictly milestone-based SPARK-BIH to serve as the front-end the validation fund or the Digital Health
and projects are closely monitored by of innovation and growth for early- Accelerator.
members of the SPARK team. stage ideas. The Inventors for Health
Program has been developed in close
Teams selected for funding by the cooperation with their partner, the Dr. Tanja Rosenmund
validation fund enter the SPARK- Stiftung Charité. The program focuses on Program Manager & Manager
BIH program. Here, projects are developing breakthrough ideas in health Validation
supported with mentoring, advice, and and is currently made up of 2 phases
an extensive network to accelerate the – a development and funding phase.
translation of their academic invention Through a series of hands-on bootcamps,
into a marketable product to the benefit inventors identify and elaborate on their
of patients and society. Furthermore, ideas, develop entrepreneurial skills,
5SPARK-BIH is part of the global SPARK network
Over 50 different institutions have established or are developing their own versions of the SPARK program. The
mission is to improve human health by uniting academics and industry experts around the globe to generate an
efficient, effective and exciting pathway for translation of academic discoveries and innovative solutions through
education and project support.
6SPARK-BIH is part of BIH Innovations
SPARK-BIH and the validation fund are part are part of BIH Innovations, the joint technology transfer office of
the Charité and the BIH. BIH Innovations consists of four parts enabling the translation and commercialization of
academic projects. SPARK-BIH is closely working with the patents & licensing team to in order to protect and exploit
innovations.
Patents, licensing, law Strategic cooperations
and startup consulting Forming and managing-
Identifying, protecting strategic co-operations
and commercializing with academia and industry
assets (with key partner:
Ascenion)
Validation Fund/
Digital Labs
SPARK-BIH
Translating digital health
Translating medical solutions to patients/
inventions (drugs, diag- market (incl. Digital
nostics, medtech) into Health Accelerator)
products and solutions
to benefit patients
7How do SPARK-BIH and the validation fund work?
SPARK-BIH and the validation fund were created to support academics in translating their inventions into novel
therapeutics, medical devices and diagnostics that address unmet medical needs.
SPARK-BIH and the validation fund are part of BIH
Innovations, the joint technology transfer of BIH and SPARK-BIH provides
Charité. mentoring, funding,
external expertise and
The purpose of SPARK-BIH and the validation fund is education.
to support academic projects with funding, mentoring,
Funding
advice and education to accelerate their translation Mentoring
The BIH validation
for the benefit of patients and society. Project managers fund offers strictly
support SPARK teams milestone-based
Our support is reflected in translationally relevant with regular meetings funding since 2018 and
preclinical data, sound clinical trials, new IP, novel life to identify needs is divided into two
and experts, access tracks for different
science startups and industry partnerships as well as progress and guide the project maturity. Calls
licensing agreements. project development are announced
annually.
By applying for funding within the validation fund
to enhance the translatability of your project you
automatically profit from the SPARK-BIH mentoring and
education program.
Project proposals are evaluated on the basis of several Advisor Education
aspects, the most important being: (1) addressing an Experts advise
Public educational
unmet medical need, (2) the potential for translation into SPARK teams on topics
seminars introduce the
such as HTS, medicinal
the clinics. drug and device devel-
chemistry, manu-
opment process, discuss
Refunding mechanisms (e.g. shares) secure that in case of facturing, clinical trials,
IP and business
regulatory and
(entrepreneurship) -
commercial successes a part of the revenues will be used business
relevant topics
development.
for future development programs.
8How does SPARK-I4H work?
The Inventors for Health (I4H) Program is a pilot program that has been developed together with the Stiftung
Charité and addresses scientists with very early projects and ideas.
Traditionally, the barriers-to-entry for medical innovation,
research and development have been high. The I4H pilot program
is democratizing that process and allowing for citizens and
patients to co-develop solutions together with scientists and medical
professionals. Phase I Phase II
Unlike traditional incubation programs focusing purely on later I4H Bootcamp I4H Grant
commercialization, the I4H program focuses on developing
breakthrough medical innovations by developing the people behind
the ideas through skill-building, mindset change, and exposure to a
Problem –
network of innovators and medical change-makers.
Solution Funding
Accordingly, the I4H pilot program tests a novel program design. Exploration
In future it is planned to continue key components of the program
as scouting mechanisms to identify and develop early ideas.
Subsequent funding might be realized by established funding Product –
Prototype
programs such as SPARK-BIH and the validation fund or the Digital Market
Refinement
Health Accelerator. Exploration
Currently, I4H is made up of two phases: In the first phase,
inventors develop their idea(s) rapidly through design-based
approaches, with help from experts from other disciplines. In the Scale and
MVP
second phase, funded teams advance their ideas to minimally Robustness
viable products (MVP) for testing and further development, making
use of SPARK-BIH.
9SPARK-BIH: Maturity Pipeline of funding opportunities by Stiftung Charité
and BIH Validation Fund
Support by SPARK-BIH mentoring & education program
BIH Validation Fund by
BIH Validation
Fund Track 2
Projects in a more
mature phase which are
I4H Incubator of BIH Validation typically patented and
Fund Track 1 need further supporting
I4H Grant Projects in an early phase data and additional
Stiftung Charité of development which are development. Examples
Outstanding projects clearly later than pure are safety, dose-finding,
I4H Bootcamp which took part in phase research projects. Visible, medical chemistry,
Phase I with interactive I are selected for fund- first proof of principle regulatory strategy,
PROJECT STATUS
Bootcamps for ing to develop a strong data which need to be clinical trial design
hands-on skill and PoC. Selected inventors validated and completed and further to be
mindset development will receive up to 50% and are usually used to translated into the clinic.
with a focus on salary support and a file a patent. Funding Funding above €50,000,
entrepreneurial and maximum of €50.000 in amount is up to €50.000, with a funding duration
design-lead approaches. project support. funding duration is 1 year. is 2 years.
Maturity & translatability of projects
10SPARK-BIH
in Numbers
SPARK projects from 2015-2019
11
11Number of SPARK projects from 2015-2019
12Translational Success of SPARK projects from 2015-2019
of our SPARK teams stated that of our SPARK Pharma teams were
71% the SPARK program was crucial to
advance the translational pathway 80% able to proceed their preclinical
development program to the next
of their project stages
100%
of our SPARK medtech
Follow-on applied research funding and IVD teams were able to
14 programs were acquired by our
SPARK teams amounting to more
proceed their medical device or
IVD development program to the next stages
than 10,5 Mio EUR
All of them also recruited an expert in CE
certification or started with the technical doc-
umentation and /or QMS 13485 establishment
11 SPARK teams have filed patents
during or after participation in SPARK
for their product
3 SPARK teams have an option contract
or term sheet for potential licensing of
87 Accelerator or Bootcamp programs
were started by our SPARK teams
their patent ongoing
1614 Prices or Awards have been won by
our SPARK teams
7 SPARK teams have founded or plan
to found a startup
87
of our SPARK teams prepared
clinical trials for their project or
8 SPARK teams have had were able to continue and improve
cooperations with industry 14 the running clinical trials
*All numbers are based on a survey that was submitted by 21 SPARK teams that started funding until 2018 as well as consultation of the patent team of BIH
innovation
13Biomedical Fields and Areas of SPARK funded projects from 2015-2019
14Statistics on SPARK organized Educational Seminars 2015-2019
“I am here to get or keep in touch with “entrepreneurial” ideas and find inspiration for translation from science
to business. It is the first time I participated and very much liked the format.“ – Attendee in Forum in 2017
50 33
SPARK organized Different topics on
educational seminars drug & device
from 2015-2019 development
Overall satisfaction
97%* with the educational
lecture series from
2015- 2019
97
Participants in the Speakers giving a
>1100 educational lecture presentation from
series from 2015- 2019 2015 - 2019
*Numbers are based on evaluations submitted by seminar attendees for 79 speakers at 40 different educational seminars in the years 2015-2019
15“In the beginning I considered SPARK just another program among all those university-based
supporting initiatives. I hoped for some financial support but not more. However, it turned
out to provide something much more valuable than money. SPARK provides multi-level
competence, bringing in the right experts at the right time. In our case, this was not only
the critical mind of other SPARKees, but also clinicians, advisors and business experienced
people, pushing our project to a much higher level. Giving us a special pre-hearing during the
Go-Bio application was extremely helpful. The payless support by a team of experts, tailor
made to our needs was a so far unique experience for me. Personally, I learned a lot, starting
from being a purely academic person, I meanwhile got a sense of how business people think
and regulatory boards work. Still there are a lot of things I need to learn to survive in this new
environment. I highly appreciate to have SPARK on my side on this journey. “
Prof. Christoph Schwarzer
16SPARK-BIH
Projects
Our newly funded & mentored SPARK Teams of 2019
17“The SPARK program was instrumental
in supporting the MyoPax idea from a basic research project into a solid
GMP-compatible product presently under preclinical testing. Without
SPARK, any translational effort into the clinic would have ceased two
years ago.“
Prof. Simone Spuler
18A novel peptide to regenerate the central nervous system
PRINCIPAL INVESTIGATOR:
Dr. Sarah-Christin Starossom Pharma - small molecule
Prof. Dr. Friedemann Paul Neurology
Charité
SUMMARY PROJECT GOALS
The team has identified a novel mechanism with
• Develop a patentable therapeutic agent
pro-oligodendrogenic and remyelinating capacity.
Based on that mechanism the project aims at • Test in vitro dose-response
developing a novel therapeutic option for multiple • Submit invention disclosure
sclerosis. Although myelin and oligodendrocytes
Track 1
are thought to be the prime target for autoimmune
attacks in multiple sclerosis (MS), there are LONG-TERM GOALS
currently no FDA approved drugs targeting the
induction of endogenous remyelination and • Novel drug therapy for Multiple
oligodendrocyte repair. The therapeutic induction of
sclerosis
oligodendrogenesis and/or remyelination is a highly
unmet need for MS and other diseases of the CNS. • License to Pharma
19Validation of anti-cancer agents in patient-derived canceroids
INVESTIGATOR: Pharma - small molecule
Prof. Dr. Reinhold Schäfer
Charité Oncology
SUMMARY PROJECT GOALS
The project aims to validate modulators of a
• Validate drug candidates in
transcription factor as agents against cancer. The
validation will be conducted in patient-derived patient-derived canceroids
canceroids. About 1/3 of all cancers are mutant • Submit invention disclosure
RAS-driven and cause up to 1 million deaths each
Track 1
year. Novel therapies targeting signaling effector LONG-TERM GOALS
proteins downstream of RAS are already in clinical
use, however with uncertain long-term effects. The • Start an investigator initiated trial
approach may provide a new avenue for anti-RAS
for cancer patients based on
therapies.
repositioned drugs
• License to Pharma
20“SPARK put me on track to focus on the goal to develop a drug and ac-
quire the competencies needed.“
“SPARK helped me to put my exploratory, basic scientist mind to the
sideline for a while, to focus on bringing the results of our research to
patients and market.“
Prof. Regine Heilbronn
21Exploring a novel therapeutic target in cystic fibrosis
PRINCIPAL INVESTIGATOR: Pharma - small molecule
Dr. Anita Balázs
Charité Pulmonology
SUMMARY PROJECT GOALS
The project aim is to validate and pre-clinically
• Validate and pre-clinically test drug
test candidates previously identified as potential
modulators of an alternative pathway to circumvent candidates for CF
the primary ion transport defect in cystic fibrosis • Submit invention disclosure
(CF). CF is a life-limiting disease caused by mutations
Track 1
in the CFTR gene. Although highly effective CFTR
modulators are emerging, ~10-15% of patients will LONG-TERM GOALS
not benefit from these therapies, while the high price
of these drugs prevents access in many countries. • Novel drug therapy for cystic fibrosis
There is an unmet need to develop alternative
and other muco-obstructive lung
therapy strategies to bypass CFTR dysfunction and to
restore epithelial ion transport in CF. diseases
• License to Pharma
22A novel solution for a total artificial heart
INVESTIGATORS:
Dr. Marcus Granegger Medtech
Tim Bierewirtz Cardiovascular Devices
Charité
SUMMARY PROJECT GOALS
• Build a functional model /
The team aims to develop a functional prototype of
an implantable total artificial heart (TAH) for the prototype
treatment of end-staged heart failure in pediatric • Perform in vitro validation
and adult patients. Available TAHs are risk prone • Submit invention disclosure
Track 1
regarding reliability, blood damage and thrombus
formation. The novel concept promises superior
LONG-TERM GOALS
performances by means of reliability, implantability
and hemocompatibilty.
• Novel cardiologic medical device for
end-staged heart failure
• License to medtech or startup foundation
23Development of a stapler for solid organs
PRINCIPAL INVESTIGATOR: Medtech
Dr. Panagiotis Fikatas
Charité Surgery
SUMMARY PROJECT GOALS
The aim of the project is the final development and
• Build a functional stapler prototype
validation of a novel surgical stapler that allows the
minimal-invasive, wedge-shaped resection of solid • Perform validation tests in “dry-lab”
organs. Current solutions for solid organ resection are
limited and carry the risk for intra- or postoperative LONG-TERM GOALS
Track 2
bleeding and are associated with a high probability
of fistula development. The novel stapler allows • Startup foundation
to cut solid organs safely and with reduced risk of • CE certification as a medical device
complications.
24A novel advanced therapy medicinal product (ATMP) to treat solid tumors
PRINCIPAL INVESTIGATOR: Pharma - ATMP
Prof. Dr. Gabriele Pecher
Charité Oncology
SUMMARY PROJECT GOALS
So far, there is no cure for instance for patients with
• Complete the preclinical
metastatic breast or pancreatic cancer and new
therapies are urgently needed. The project aims development of the ATMP
to develop a novel ATMP for the therapy of solid • Prepare phase I / II clinical trial
tumors. The group will use the innovative ATMP in • File novel patent
Track 2
order to generate optimized immune cells to fight
the immunosuppressive microenvironment of solid
LONG-TERM GOALS
tumors. The ATMP will be validated and preclinical
testing will be accomplished.
• Perform phase I / II clinical trial
• License to Pharma or startup
foundation
25A novel gene therapy for treatment of aggressive B cell lymphoma
PRINCIPAL INVESTIGATOR:
Prof. Dr. Antonio Pezzutto Pharma - ATMP
PD Dr. Antonia Busse Oncology
Charité
SUMMARY PROJECT GOALS
The project seeks to complete the preclinical
• Complete the preclinical
characterization of a human-derived T cell receptor
(TCR) that selectively recognizes a specific point development of the ATMP
mutation of the protein MyD88. This specific mutation • Prepare phase I clinical trial
is a key oncogenic driver event in around 20% of • Establish industry partnership
Track 2
patients with an aggressive variant of Diffuse Large
B cell lymphoma, and in around 50% of patients with
Primary CNS lymphoma. Both patient populations LONG-TERM GOALS
have a poor prognosis, and only limited therapies
are available especially for the elderly and patients • Perform phase I clinical trial
with severe comorbidities. Adoptive T cell therapy
• License to Pharma
with MyD88-specific T cells would represent a truly
tumor-specific therapy, being much more selective
than CAR T cells or immune checkpoint inhibitors.
26GrOwnValve – Anchoring mechanism for a personalized, autologous heart
valve for children
PRINCIPAL INVESTIGATOR: Medtech
PD Dr. Boris Schmitt
Charité Cardiology
SUMMARY PROJECT GOALS
The aim of the project is the production and testing
• Perform preclinical testing of
of an anchoring mechanism of a personalized,
autologous heart valve for children enabling growth anchoring mechanism together with
in a once-in-a-lifetime point-of-care minimally the valve
invasive implantation. The novel anchoring • Prepare phase II clinical trial in
Track 2
mechanism facilitates placement of the valve without children
hindering growth of valve and vessel. For babies
born with a congenital heart valve defect there is no
dedicated child valve on the market. Instead they LONG-TERM GOALS
often receive xenogenic animal valves which degrade
over the following years urging for risky open-heart • Perform phase II clinical trial in
re-surgery. children
• Start-up foundation
• CE certification as a medical device
27In vivo validation of a novel class of pain medication
PRINCIPAL INVESTIGATOR:
Dr. Viola Seitz Pharma - small molecule
Prof. Dr. Christoph Stein Pain
Charité
SUMMARY PROJECT GOALS
The project seeks to complete external validation of
• External validation of preclinical in
in vivo data on NFEPP, a novel compound that has
demonstrated potent pain relief without addiction vivo studies
potential in initial experiments. A patent has been • Secure follow-on applied research
filed and results were published in Science in 2017. funding
Track 2
Although pain research has identified a plethora
of targets, no truly innovative analgesics have
reached the market in the past years, mostly due LONG-TERM GOALS
to low efficacy or severe side effects. This leaves a
significant unmet medical need for novel, safe and • Further develop under CMC and GMP
effective compounds with reduced side effect burden
conditions
and abuse liability.
• Test safety & toxicity of NFEPP
• Perform phase I/IIa clinical trials
• License to Pharma
28SPARK-BIH
Projects
Track 2
Our SPARK Teams that started funding & mentoring
in 2015 - 2018
29Development of a platform for the isolation of T cell receptors for cancer
immunotherapy
PRINCIPAL INVESTIGATOR:
Dr. Felix Lorenz, Dr. Julian Clauss, Dr. Inan Pharma - ATMP
Edes, Prof. Dr. Wolfgang Uckert Oncology
Max Delbrück Center for Molecular Medicine
SUMMARY PROJECT ACHIEVEMENTS
The project is designed to develop a high throughput
DURING & AFTER SPARK
platform to identify T cell receptors (TCRs) specific
for cancer antigens to target new patient groups • Robust platform established to
with TCR immunotherapy. Preliminary studies not develop >30 isolated TCRs
only demonstrated the feasibility of the strategy, • Multiple patents filed from 2015-2019
but also identified two novel TCRs. Patents covering
2015
• Winner at life sciences and
these TCRs and the platform were previously filed at
healthcare startup accelerator
the European Patent Office. The team is setting up
a startup (called Captain T Cell) and has acquired OneStart in 2016
follow-up funding to further pursue the strategy • Jury price at BioVaria showcasing
and develop TCRs for the use in patients as well event for life science technologies in
as increasing the output of this novel and unique 2017
platform. A service lab (Helmholtz Innovation Lab)
• Total follow-on pre-seed funding of
has been established in parallel at the Max-Delbrück-
Center. 4 Mio. € until 2020
• Startup planned for 2020
30“SPARK was the starting point for my whole team to start thinking how we
can translate our research results in developing drugs for patients.“
“I was so excited to participate in the SPARK mentoring program, because
it provides a completely new perspective on the clinical translation of
projects. For scientists it is so important to get insights into the process of
translating basic research findings into treatments. These insights
provided by the SPARK program definitely shaped my way of thinking
about current and future scientific projects.”
Dr. Felix Lorenz
31“SPARK enabled us to translate an initial mechanistic idea into a
successful preclinical drug development project.“
“SPARK is a great initiative to de-risk innovative projects and to
enhance translational research from bench to bedside. SPARK provided us
with excellent partners, pushed our project and helped a lot in making
decisions and focusing on the next steps.”
PD Dr. Karoline Krause
32New treatment strategies by targeting the inflammasome
PRINCIPAL INVESTIGATOR: Pharma - small molecule
PD Dr. Karoline Krause
Charité Dermatology
SUMMARY PROJECT ACHIEVEMENTS
The project has identified several inhibitors of
DURING & AFTER SPARK
inflammasome activation in a high-content screen.
Inflammasome activation is a hallmark of several • Identified several lead candidates
monogenic and complex systemic auto-inflammatory • Extended indication & initiated
diseases for which only few standard therapies exist. collaboration
In addition, inflammasome activation plays a central
2015
• Preclinical proof of concept in human
role in the pathogenesis of additional diseases such
skin models
as contact dermatitis. The identified inhibitors are
evaluated for their use in the different indications. • Patent filing planned for 2020
One compound has the potential to be repurposed. • Started writing Investigator’s
brochure
• Started partnering with Pharma
concerning novel chemical entity
33Gene therapy for the treatment of temporal lobe epilepsy
PRINCIPAL INVESTIGATOR:
Prof. Regine Heilbronn Pharma - small molecule
Prof. Christoph Schwarzer Neurology
Charité & Medizinische Universität Innsbruck
SUMMARY PROJECT ACHIEVEMENTS
The project aims at developing a gene therapy for the
DURING & AFTER SPARK
treatment of drug-resistant focal epilepsy. An adeno-
associated viral (AAV) vector will be delivered to the • Patents filed in 2016
epileptic focus, re-expressing a neuropeptide that • Preclinical Proof-of-concept in vivo
will be released in an activity-dependent manner, i.e. and human brain tissue ex vivo in
in periods of high neuronal activity which precedes
2015
2016
the onset of a seizure. Suppression of neuronal
• Secured GoBio funding of 3.9 Mio. €
excitability thereby suppresses the epileptic event.
Strong proof of concept data in mice and rats have in 2018 for 3 years
supported the feasibility of this strategy. The team • GMP production in preparation
is setting up a startup and has acquired follow-up • Startup planned for 2021
funding to further pursue the strategy and develop
the gene therapy for the use in patients.
34“When first invited, I came for money. Now I come for priceless
expertise and multi-level support.“
Prof. Christoph Schwarzer
35FiXatas - Ready-to use surgical knots
PRINCIPAL INVESTIGATOR: Medtech
Dr. Panagiotis Fikatas
Charité Surgery
SUMMARY PROJECT ACHIEVEMENTS
In the project a device and method for the generation
DURING & AFTER SPARK
of extra corporally pre-tied surgical knots has been
developed. The device consists of a yarn carrier with • Patent granted in 2018
a pre-tied but still open knot ready to use during • Project developed from invention to
surgery. It is easy to use even by non-surgeons marketable product
without special training. Knots produced are stronger
2016
2016
• Winner of the Ethicon Future Award
and more stable than other sliding knots and tying
2016
is faster. Potential user groups have been extended.
The first use field will be endoscopic surgery where • 3rd Place of PROFUND “Research to
tying knots is very challenging due to limitations Market Challenge 2017”, 2nd Place at
in space and the visual field. Several patents and BPW 2018 contest, 2nd Place at YES!
designs have been filed. The team has founded a Delft Pitching 2019
startup in early 2020.
• Started negotiations with medtech
• Startup founded in 2020
36Lead optimization of STOML3 inhibitors for the treatment of neuropathic pain
PRINCIPAL INVESTIGATOR:
Pharma - small molecule
Prof. Dr. Gary Lewin,
Dr. Christiane Wetzel Pain
Max Delbrück Center for Molecular Medicine
SUMMARY PROJECT ACHIEVEMENTS
In this project a small molecule inhibitor for STOML3
DURING & AFTER SPARK
will be developed to treat neuropathic pain. The
protein is required for the transduction of pain signals • Preclinical proof-of-concept in 2016
in peripheral pain receptors. STOML3 expression • Patent filed in 2016
is upregulated after nerve injury in sensory fibers • Publication: 2017
making it a great target. In a high throughput screen
2015
• Secured Helmholtz Validation Fund
several inhibitors of STOML3 oligomerization and
in 2016 for further development and
thus (mechano)transduction were identified. In vivo
proof of concept has been achieved in two mouse optimization of lead compounds
models for neuropathic pain. Neuropathic pain candidates
is a condition caused by nerve damage or disease
affecting the nervous system. In half of the patients
pain relief cannot be achieved by current treatment
options.
37Validation study for cervical HPV and dysplasia screening test
PRINCIPAL INVESTIGATOR: In Vitro Diagnostic
PD Dr. Andreas Kaufmann
Charité Oncology
SUMMARY PROJECT ACHIEVEMENTS
The team has developed a diagnostic test for
DURING & AFTER SPARK
cervical HPV infection and dysplasia detection with
high sensitivity, specificity as well as a high positive • Developed a multiplexed
predictive value. The initial use is in triaging of quantitative mRNA-based test
equivocal screening findings. Current tests like combining HPV and biomarker
cytology and PCR-based HPV testing either lack
2016
2016
expression
diagnostic accuracy or require a biopsy in follow up.
• Developed algorithm to predict
After patenting, the team is currently performing a
clinical study. In the future, CE certification of the disease stage
test and accreditation of a service lab are planned to • Patent filed in 2019
bring the test to the patients. Cervical cancer is the • Study planned and initiated
second most common cancer in women living in low
and middle income countries with more than half a
million new cases in 2018. Due to lack in standard
screening procedures the test could be used there
as a screening tool.
38“SPARK has fostered an outside view on our IVD product helping to
consider critical aspects of development and marketing. “
PD Dr. Andreas KaufmannInhibitors of ribosome assembly
PRINCIPAL INVESTIGATOR: Pharma - small moelcule
Dr. Rainer Nikolay
Charité Infection
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
In this project it is planned to develop a new class
of antibiotics, based on the inhibition of prokaryotic • HTS was performed at the FMP
ribosome assembly. According to the WHO antibiotic • Due to low specificity of the
resistance is a global threat. The team has developed screening assay hit candidates could
an in-vivo screening assay based on reporter strains,
2016
2016
not be identified
where large and small ribosomal subunits have
• Advise to improve the screening
been tagged with red or green fluorescent proteins.
A disturbance in subunit assembly can be detected assay & evaluate alternative
via the fluorescent ratio. Based on this fluorescence- approaches (e.g. structure-based
based reporter assay, a high throughput screen was design)
performed to identify small molecule inhibitors that • Identified hit compounds in in-silico
specifically interfere with the assembly of either the
structure-based design methods in
large or the small ribosomal subunit and thereby
inhibit bacterial growth. collaboration with AG-Wolber (FU
Berlin)
• Hit compounds are being tested in
several assays.
40We build muscles– the human muscle stem cell
PRINCIPAL INVESTIGATOR:
Prof. Dr. Simone Spuler Pharma - ATMP
Dr. Verena Schöwel Stem cells – Muscle repair
MDC & Charité
PROJECT ACHIEVEMENTS
SUMMARY DURING & AFTER SPARK
Muscle wasting and weakness are leading symptoms • Preclinical Proof-of-concept,
of a wide variety of diseases. The entire muscle is preclinical safety ongoing, PEI
affected or only single muscles do not function,
yet with dramatic impairment of life quality and scientific advice meetings
life-threatening consequences. Muscle diseases are • Planning of phase I/IIa clinical trial
2016
untreatable. In Europe alone, over 6 million citizens • Follow-on funding acquired:
are affected. The team MyoPax develops an innovative Helmholtz Enterprise 2018-2019, IBB
autologous muscle stem cell therapy to treat muscle Coaching Bonus 2019, Translatorik
wasting. The team’s technological innovation
enables highly standardized manufacturing of program of the Else Kröner-Fresenius
pure, native and highly regenerative muscle stem Foundation 2019-2020
cell populations out of small human muscle tissue • Science4Life award 2019 for “MyoPax”
specimens. This enables the team to set up multiple business concept
treatment algorithms for acquired and inherited • Charité Entrepreneurship Summit
muscle diseases. The team has acquired follow-up
funding and prepares to set up a startup company to pitch award winner 2019
clinically pursue the development of their approach • Pitch contribution at Bio-Europe 2017
to fight muscle diseases. and World Health Summit 2019
41matrix for antigen-specific secetion of plasma cells secreting pathogenic
autoantibodies
PRINCIPAL INVESTIGATOR: Pharma - small moelcule
Prof. Dr. Falk Hiepe
Charité Immunology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
Long-lived memory plasma cells secreting pathogenic
antibodies have emerged as a promising therapeutic • Completed the SPARK funding period
target since these cells are resistant to conventional successfully
immunosuppressive drugs and therapies targeting B • Winner of the Sanofi iAward 2018 &
cells. Current therapies targeting plasma cells such
2016
2016
2020
as proteasome inhibitors deplete all plasma cells
• Project projected to reach preclinical
including those contributing to humoral immunity
that may result in immuno-deficiency. Therefore, proof of concept by 2025
the group developed a concept that is able to
deplete plasma cells based on the specificity of
their secreted antibodies. The group aims to use
deplete autoantibody-secreting plasma cells in a
murine autoimmune model in order to demonstrate
functionality of the technique. The project recently
gained interest and support by industry.
42“The SPARK program was absolutely “SPARK is fundamental for
decisive to change our mindset in researchers from any field to bring an
planning and thinking of experiments idea from research to what needs to be
and research, from being limited to basic done in the business field.“
research to a holistic understanding of
the requirements of translational Dr. Alessandro Faragli
research.“
Dr. Philipp Enghard
43Flurinocyte - urine flow cytometry as biomarker for renal diseases
PRINCIPAL INVESTIGATOR: In vitro Diagnostic
Dr. Philipp Enghard
Charité Nephrology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
The project aims at developing a diagnostic assay
to quantify cellular components present in human • Data validation in different patient
urine via flow cytometry. Cells identified and groups
quantified allow the differential diagnosis of several • Established an easy-to-use sample
renal diseases. The presence or absence of specific
2016
2016
2017
2017
conservation protocol to simplify
cell types correlated to disease activity and disease
2015
the test logistics
severity. This simple urine test could be used as a
diagnostic tool, to screen patients who need a renal • Currently validating marker and
biopsy, monitor treatment and predict outcome. The sample logistics in two multi-center
assay could become a helpful tool in the clinic when studies on renal
a quick primary assessment is required to define diseases
subsequent clinical workup and may enable a more
personalized treatment.
44Prediction and prevention of congestive events in heart failure patients
INVESTIGATOR: Digital Health
Dr. Alessandro Faragli
Charité Cardiology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
Heart failure (HF) represents the leading cause of
hospitalization worldwide. Because of the difficulty in • Risk predictors identified
treating this chronic disease, re-hospitalizations are • First algorithm designed
associated with high cost for the healthcare systems,
accounting for €28 billion per year in Europe only. LONG-TERM GOALS
2016
2017
2017
The team aims at building an algorithm to predict and
prevent congestive events in heart failure patients. • Perform clinical study to determine
They have identified risk predictors and created an
accuracy of prediction algorithm
algorithm. At the moment, the team is performing a
database analysis on existing cohorts of HF patients • Submit invention disclosure
to build the first risk prediction model. • License to medtech or startup
foundation
45Novel drugs strengthening endogenous immuno-regulatory processes
PRINCIPAL INVESTIGATOR:
Pharma - small molecule
Dr. Stefan Frischbutter
Charité & German Rheumatism Research Center Immunology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
The balance in the immune system between
suppression or activation of immune responses • Hits from drug screen have been
is highly complex, very tightly regulated and validated and immune responses
fine-tuned with multiple cell types orchestrated. were partially dissected
If this fine-tuning is out of balance, diseases such
2016
2016
2017
2017
• Target validation and drug
as autoimmunity occur. In this project the team
2015
development under way
is focusing on the suppressive capacity of the
immune system and is evaluating the use of drugs • Major hurdles have been identified
to bring this arm back in balance. The team has in pursuing the idea into the clinic
developed a high-throughput-screening platform
and identified already several bioactive molecules
with re-balancing capacity. The team has validated
these drug candidates in primary human immune
cells and assessed the resulting immune reaction
and potential impact on disease.
46BodyTime - A new diagnostic tool to assess the internal clock
PRINCIPAL INVESTIGATOR: In vitro Diagnostic
Prof. Dr. Achim Kramer
Charité Other
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
The circadian clock is a biological program that
structures physiology and behaviour according to • Identified core set of time-telling
the time of day. It is active in practically all cells of genes
our bodies. The circadian clock is thus a cell-based • Patent filed in 2018
program that is essential to health and well-being.
2016
2017
2017
• Developed a robust assay and
The team has developed a new diagnostic tool to
2015
predictive algorithm with 30 min
probe human internal time and rhythm using a single
blood sample. It has utility in defining the correct accuracy
time of day for drug dosing, in order to achieve the • Follow-on applied research funding
least adverse effects. Of note, >50% of the top selling by BIH Digital Health Accelerator
drugs target clock-controlled genes and thus likely • Started beta-testing with different
have specific time of day effectiveness. This solution
patient cohorts in 2019
can therefore offer value in reducing side effects
as well as helping with sleep disorders or work • Startup foundation planned in late
performance. 2020
47“Before entering the SPARK “We are very thankful to SPARK
program, we had many translational for laying in front of us the whole
paths in mind. Now we have found spectrum of expertise in the field of drug
the most promising one.“ development and giving us access to
role-models in the field conveying that
Dr. Stefan Frischbutter “we can do it” as well.“
Prof. Dr. Markus Schülke“Through the educational and entrepreneurial forum I profited from
experts and was trained in bringing therapy into clinic.
I also got insights into hurdles, developmental processes,
regulations that other SPARK projects are dealing with.
The financial support allowed us to carry on the translational process
of our project. Brainstorming for solving unexpected reactivity of our
reagent was finally crucial to save the whole
project.“
Dr. Simone RheinTCR gene therapy of CD22-positive B cell malignancies
PRINCIPAL INVESTIGATOR:
Dr. Simone Rhein Pharma - ATMP
Prof. Antonio Pezzutto Oncology
Charité
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
In this project, the goal is to generate a TCR for
gene therapy of B cell malignancies by targeting • novel CD22-specific TCRs identified
the B cell antigen CD22. CD22 is a good alternative • patent filed in 2018 (second TCR
target to CD19- and CD20-directed therapies as added in 2019)
these therapies often lead to downregulation
2018
2016
or loss of the antigen. CD22-targeted CAR T cells
induce remission in B-ALL that is naive or resistant LONG-TERM GOALS
to CD19-targeted CAR immunotherapy. The clinical
success however is hampered by downregulation of • Demonstration of superiority over
surface CD22 expression upon CAR treatment, which
CD22 CAR T cells
greatly reduces efficacy. Since TCRs do not depend
on antigen surface expression, these patients could • Perform phase I clinical trial
benefit from TCR T cell therapy. A newly generated • License to Pharma
TCR candidate is being tested for off-target toxicity
and will be compared to CD22 CAR T cells. A patent
has been filed on the CD22-specific TCR.
50Drug discovery for mitochondrially inherited Leigh syndrome (MILS)
PRINCIPAL INVESTIGATORS:
Prof. Dr. Alessandro Prigione Pharma – small molecule
Prof. Dr. Markus Schülke Neurology
MDC & Charité
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
The team has developed a novel assay system
based on patient-derived induced pluripotent stem • Identified and validated compound
cells (iPSCs) to identify compounds for treating class for treatment of Leigh
Leigh syndrome. Using this assay, a class of drugs syndrome
applicable for repurposing that restore the cellular
2018
• Performed compassionate treatment
disease phenotype has been identified. The team
• Plan to prepare phase I/II orphan
has initiated a compassionate use treatment for
a terminal ill patient. The patient has recovered drug repurposing trial
significantly. Based on these results a clinical
study is planned. Leigh syndrome is a rare severe
mitochondrial disease affecting children where
treatment options are lacking.
51“SPARK is a great platform to learn about the drug development
process and to think as a translational researcher.
It exposed us to topics which we as basic scientists typically do not have
on our daily agenda but which are vital for translation, such as intellectual
property and requirements for clinical trial design.
Advice from other SPARKees was helpful in streamlining the way towards a
drug and to identify important checkpoints within preclinical
validation.“
Prof. Dr. Chiara Romagnani
52Peptide-based vaccination targeting innate responses against viral
infection and cancer
PRINCIPAL INVESTIGATOR: Pharma – small molecule
Prof. Dr. Chiara Romagnani
Charité & German Rheumatism Research Centre Oncology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
The immune system has evolved different strategies
to prevent and fight cancer and infections. Natural • Co-stimulants assessed for vaccine
Killer (NK) cells are an innate immune cell type development
able to kill tumor and/or infected cells. The team • Partners for development of vaccine
has identified a specific peptide whose expression
2018
identified
is shared by cytomegalovirus and certain tumors.
• Clinical indication and potential
The project aims to exploit this immunological
phenomenon by using the peptide as a vaccine clinical partners identified
directed against tumors and cytomegalovirus, • In-vivo proof-of-concept ongoing
simultaneously. The advantage of this approach is
that cancer patients can benefit from this boost for
the immune system that is ready not only to fight
cytomegalovirus but also relapsing tumors.
53Molecular imaging of biofilm infections - Validation of FISH controls for
automated endocarditis diagnostics
PRINCIPAL INVESTIGATORS:
PD Dr. Annette Moter In Vitro Diagnostic
Dr. Judith Kikhney Infection
Charité
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
Fluorescence in situ hybridization (FISH) is a
molecular technique, which allows identification • Design and validation of controls
and visualization of microorganisms within tissues. • Developed a sample tracking
Currently, the daily diagnostic use of FISH is software
restricted to highly specialized laboratories because
2018
• Currently developing
it involves not only high-level of expertise, but also
semi-automated digital image
many hands-on steps, time-consuming microscopy,
laborious annotation and documentation of FISH analysis for detection of bacteria in
images and is lacking standard high quality controls. histological sections
In this project diagnostic use of FISH in daily routine • Developed an intelligent
for endocarditis diagnostics is tested by automating image handling archiving and
the full process of this technique. The group is
documentation system
focusing on multiple aspects of this diagnostic
procedure – with one emphasis on the generation of • Currently testing entire platform in
solid and validated routine positive controls. routine diagnostic and comparing
the ‘hands-on’ with the automated
FISH (within the BMBF-funded iSOLID
consortium)
54“Without financial support, the
project would not have been possible and the fantastic SPARK
team helped us to achieve tremendous progress in a structured
and well-focues way.“
Dr. Kostja Renko
55Image-based Support of Minimal-Invasive Mitral Valve Repair
PRINCIPAL INVESTIGATOR: Digital Health
Prof. Dr. Anja Hennemuth
Charité & German Rheumatism Research Centre Cardiology
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
About 7000 isolated mitral valve surgeries are
performed in Germany every year. Mitral valve repair • Started development of software
(MVR) is superior to valve replacement. Successful modules for image fusion and
repair does not only lead to better survival but also integrated visualization and
better quality of life and avoidance of anticoagulants.
2018
interaction
However MVR success rates strongly correlate with the
• Started setting up quality
experience of the surgeon as MVR is difficult to learn
due to differences in pre-OP images of the moving management and documentation
heart and during the operation (or during surgery). system
This indicates the need for a better intraoperative • Initiated collaborations to specify
decision support. The team works on the application user needs and interface questions
of image-based surgery planning and image-based
• Initiated industry collaborations for
navigation with different modalities. This could help
the surgeon to accurately consider anatomical and clinical integration
dynamic properties of the valve during surgery. • Acquired BMBF funding together with
industry partner
56Predicting post-operative complications in real-time
PRINCIPAL INVESTIGATOR:
Digital Health
Dr. Alexander Meyer
Prof. Dr. Volkmar Falk Cardiology
Charité & DHZB
SUMMARY PROJECT ACHIEVEMENTS DURING
& AFTER SPARK
The large number of concurrent patient data in
critical care units goes well beyond the capacity of the • Prototype ready including user interface
intensive care physician and may lead to treatment and client-server infrastructure
delays or clinical errors. The team applies deep
machine learning methods in a critical care scenario • Business plan completed
2018
2018
to provide timely and highly accurate decision • Collected first user feedback
support to the clinical staff. They have developed a • Completed team
set of forward-facing real-time prediction models for • Started recruiting partner hospitals
severe post-cardiothoracic surgery complications. • Further refined and improved bleeding
Primary focus is the prediction of postoperative
bleeding. model
• Started to work towards regulatory
approval with experts
57Inhibition of thyroid hormone inactivation for cancer treatment
PRINCIPAL INVESTIGATOR:
Pharma – small molecule
Dr. Kostja Renko
Prof. Dr. Lutz Schomburg Oncology
Charité
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
An initial compound library screen led to a preliminary
list of hormone modulators. The compound target • Verification and characterization of
reportedly plays a role in different cancer entities. >50 compounds from an HTS approach
During the SPARK funding period, drug candidates • Further testing of selected, specific
were characterized for specificity, potency and
2018
candidates on intact cells
on-cell effects. Furthermore, in silico drug design was
• Ongoing validation of candidates in
started to predict improved candidates. Experimental
approaches to verify the reported beneficial effects cancer cell lines
in a cancer cell line completed the overall strategy. • Plan of strategic cooperations with
Future plans include strategic cooperation with oncologists
oncology experts.
58Prevention of paclitaxel-related Neurotoxicity
PRINCIPAL INVESTIGATOR:
Dr. Wolfgang Böhmerle Pharma – small molecule
Prof. Dr. Matthias Endres Neurology
Charité
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
Polyneuropathy is a common, potentially long lasting
and debilitating neurological side effect for cancer • Assessment and compilation of
patients treated with paclitaxel. It has been shown, available pre-clinical data
that paclitaxel binds the neuronal-calcium sensor • Completion of GAP analysis
1 protein which causes calcium dyhomeostasis in
2018
• Consultation of BfArM on design
neurons. This process contributes to the development
of paclitaxel-induced neuropathy and can be of clinical study
selectively blocked by an approved on–the-market • Recruitment of clinical partners
drug that can be readily repositioned. In this project, • Assembly of trial infrastructure
the potential of co-administration of this drug with • Preparation of documentation and
paclitaxel to prevent neurological side effects is
design for phase II study
being exploited. Currently the team has designed
a clinical study, consulted the BfARM for scientific • Funding recruitment in progress for
advice and is recruiting funding for executing the phase II repurposing trial
study to assess the potential of the repositioned
drug to reduce paclitaxel-induced neurotoxicity.
59“SPARK support was essential to continue and to improve our
translational research project. Additionally it opened new horizons to
evaluate new alternative peptides for our target protein.“
Prof. Peter KühnenMC4R agonist treatment of patients with monogenic obesity
PRINCIPAL INVESTIGATOR: Pharma – small molecule
PD Dr. Peter Kühnen
Charité Metabolic Disease
SUMMARY PROJECT ACHIEVEMENTS
DURING & AFTER SPARK
Obesity is an increasing problem with immense
socioeconomic burden and personal suffering for
the individual patients. In this project the team has • Patent filed
identified a novel intracellular pathway via a known • Diagnostic screen established
receptor, how satiety is mediated in the cells. When
2018
this signaling pathway is disturbed, the patients • Identified several mutations and
experience a constant hunger feeling irrespective of epigenetic modifications within the
how much they eat. The goal is to identify patients MC4R pathway which might identify
that can benefit from modulating the signal with a patients for MC4R agonist treatment
known drug and change their hunger feeling thus • Enrolled several patients in the
reducing the weight naturally. The team has identified
patients with several mutations at respective ongoing Phase 2 investigator-
receptor signaling pathways as well as epigenetic initiated clinical trial
changes and are able to include these patients in an
investigator-initiated clinical trial.
61Combinatorial treatment for metastatic colorectal cancer
PRINCIPAL INVESTIGATOR:
Prof. Dr. Ulrike Stein Pharma – small molecule
Prof. Dr. Wolfgang Walther Oncology
MDC & Charité
SUMMARY PROJECT ACHIEVEMENTS
Colorectal cancer is the third most diagnosed
DURING & AFTER SPARK
cancer and fourth most common cause of death
world wide, metastasis being the cause of about • One patent granted, several patents
90% of deaths. Team Stein has identified two FDA pending
approved drugs that combined robustly inhibit the • First preclinical developmental steps
metastasis of colon cancer. They have initiated a first
2018
& POC completed
phase II clinical trial for one of the drugs – and are
• Phase II mono-therapy clinical study
aiming at initiating a second phase II clinical trial
for combinatorial therapies for colorectal cancer is running
(CRC). An option contract has been negotiated with a • Ongoing licensing negotiations &
biotech company, which also includes an option for planned industry partnership
an industry partnership with the Stein Team.
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