By Dennis Ford, Founder & CEO, Life Science Nation (LSN)
Every March, early-stage life science teams spend thousands of euros to attend one of Europe’s biggest partnering weeks. They show up expecting investors, deal momentum, and progress. Most leave with something else: lots of vendor and service provider meeting requests, and a shorter cash runway.
For seed, Series A, and early Series B companies, Lisbon is not a winter party celebration. It is a stress test. And the platforms you choose will either compound your progress or quietly drain your capital.
The Cost Reality No One Likes to Say Out Loud
Standard passes and bundled “week in Lisbon” packages routinely run from 3,000 to 5,000 euros per person, before flights and hotels. For late-stage companies, that may be acceptable. For early-stage teams living on grants, founder savings, or a small seed round, it is a major bet.
By contrast, RESI Europe is typically priced in the 1,500-2,500 euro range because it is built specifically for founders, innovators, and regional cohorts raising seed, Series A, and early Series B funding. The goal is not to sell access at any cost. It is to make high-quality global partnering economically accessible at a stage when every euro still must justify itself.
Here is the problem. Many founders pay the higher prices and then discover that a large share of their so-called “investor” meetings are with service providers selling you something. The badge may say partner or advisor, but the economics are reversed. The startup becomes the customer, not the one being backed.
That outcome is not accidental. It is how large conference ecosystems monetize scale.
Lisbon Does Not Create Strategy. It Exposes It.
Big conference weeks amplify whatever strategy you bring. If you arrive without preparation and focus, you get more noise, more meetings you did not need, and a bigger bill. If you arrive with discipline, targeted investors, and a follow-up system, Lisbon can work.
The problem is that most mega-events are optimized for volume, not readiness. More people. More meetings. More urgency. That model works for late-stage transactions. It fails early-stage teams.
Early-stage companies need fewer things done well:
Investors and licensing partners who write first checks
Fewer vendor-driven meetings
A way to turn first conversations into real follow-up and progress
Proof That a Different Model Works
At JPM Week in January, RESI was designed explicitly around early-stage investing. Roughly 800 companies actively seeking capital and licensing deals participated alongside more than 800 qualified investors and licensing partners from around the world.
Participation was not open-ended. Investor categories were defined. Registrations per firm were capped to protect the signal in the room. The result was not fewer meetings. It was better, more compelling meetings.
That same discipline is what matters in Lisbon.
Why the LSN Partnering Backbone Beats Scale
LSN, owner of the RESI conference series, also owns a premier database of capital investors and licensing partners in the life sciences and offers programs for de-risking early-stage assets and for preparing and executing global roadshows, as well as services like BD Assist, which actually sets up the meetings for you. RESI has five global partnering events annually.
A partnering backbone asks different questions. Are you spending time with partners who fit your stage and product? Have you reduced scientific, regulatory, and execution risk before asking for capital? Do you have a system to re-engage after the week ends? When the answer is yes, Lisbon stops being a gamble.
The Real Fight
The real battle for Lisbon is not about who has the biggest crowd or the loudest brand. It is about who is actually built for early-stage innovation and who is pricing and designing their platform around scale.
For founders, investors, and regions focused on seed, Series A, and early Series B, the smart move is to start the week with early-stage as the priority, not the afterthought, and with “investor” meaning capital and licensing partner, not a sales pitch. Plug Lisbon into a backbone that keeps working after the noise fades. That is how early-stage teams win Lisbon. And that is where the fight really is.
If You’re Coming to Lisbon
RESI Europe will take place in Lisbon with an in-person conference followed by virtual partnering, giving early-stage teams both face-to-face and online access to global investors and licensing partners at founder-level pricing. If you want your Lisbon week to start in a room built for early-stage innovation, not a room selling to you, RESI is where that week should begin.
Interview with Paula Cerqueira, VP of Scientific Strategy
Sania Therapeutics is developing a next-generation gene therapy platform focused on treating neurological symptoms driven by dysfunctional neural circuits. At RESI London, the company was recognized as a Third-Place winner in the Innovator’s Pitch Challenge and received the highest score from the judging panel, underscoring strong investor interest in its controllable and circuit-specific approach to gene therapy. In this interview, Sania Therapeutics shares its therapeutic focus, differentiated platform, and how participation in RESI has helped shape ongoing conversations with investors and strategic partners.
Caitlin Dolegowski (CD): For those just discovering Sania Therapeutics, how do you describe your company and therapeutic focus? Paula Cerqueira (PC): Sania Therapeutics is developing a new class of controllable gene therapies designed to treat neurological symptoms driven by dysfunctional neural circuits. Our platform combines localized, low-dose AAV delivery that selectively targets specific neuronal subpopulations with patient-controlled activation, allowing us to precisely modulate hyperactive neurons, improving symptoms without adversely and permanently altering normal neural function.
Our initial therapeutic target is a motor circuit disorder: spasticity. Our broader goal is to expand into additional motor and sensory indications where current treatment options are limited, invasive, or poorly tolerated.
CD: What unmet medical need are you addressing, and what differentiates your approach? PC: Millions of people live with debilitating neurological symptoms such as spasticity and pain disorders, yet existing treatments are often temporary, blunt, or invasive. Oral drugs frequently cause systemic side effects, while interventions like Botox or implanted devices require repeated procedures and provide limited relief. Despite the scale of this unmet need, there has been little meaningful innovation in this area for more than a decade.
Sania’s approach is differentiated in two key ways. First, our proprietary platform enables selective targeting of the neural circuits that drive disease using localized, low-dose AAV delivery. This approach is intended to support a safer, more sustainable, and more scalable path for gene therapy than traditional systemic delivery.
Second, our therapy is controllable. Patients can adjust the therapeutic effect using an oral activator, allowing symptom modulation over time. This puts patients in control while enabling precise and flexible therapeutic regulation.
Our mission at Sania is to bring gene therapy into everyday clinical use by meaningfully improving the lives of people living with neurological conditions. While this is an ambitious goal, for patients who struggle daily with basic activities such as holding a child, we believe this approach has the potential to be truly transformative.
CD: What was your experience participating in the Innovator’s Pitch Challenge at RESI London? PC: Participating in the Innovator’s Pitch Challenge at RESI London was an extremely valuable experience. The format encouraged clarity and discipline in how we communicated both our science and long-term vision, and the audience questions reflected a high level of engagement from investors and industry leaders.
Being recognized as a Third Place (First Place among judges in our session) winner among a strong and diverse group of companies was particularly meaningful, and it reinforced that there is a strong interest in approaches that rethink how gene therapy can be applied beyond ultra-rare indications.
CD: How has the RESI platform influenced conversations with investors or strategic partners? PC: RESI offered a valuable opportunity to present our work to a broad set of investors and strategic partners and to test our messaging with a highly informed audience. While many groups are understandably focused on later-stage opportunities, the platform helped us refine our positioning and identify areas of alignment for future conversations as the company progresses.
Following the Innovator’s Pitch Challenge, we also initiated early, informal conversations that we expect to build on as the company continues to mature.
CD: Where does Sania Therapeutics currently stand in terms of fundraising or partnerships? PC: Sania Therapeutics is currently focused on advancing its lead spasticity program and platform toward key preclinical and IND-enabling milestones, while continuing to expand the broader platform supporting multiple motor and sensory indications.
In parallel, we are building relationships with investors and strategic partners aligned with our long-term vision. As the platform matures and data advances, we expect to raise funding to support clinical entry of our lead program and the continued development of additional programs enabled by the platform, and we welcome conversations with groups interested in engaging early.
CD: What upcoming milestones are most important for the company? PC: Our near-term focus is on advancing our lead spasticity program across regulatory and manufacturing activities and initiating IND-enabling studies in 2026. Reaching that point will significantly de-risk the program and position us well as we move this innovative approach toward the clinic.
In parallel, we are making meaningful progress on platform development to support expansion into additional motor and sensory indications. A key goal for the team this year is to validate our first sensory capsid in vivo, leveraging the same delivery and control principles demonstrated in our lead program.
Applications are now open for the Innovator’s Pitch Challenge at RESI Europe. Life science and health tech companies seeking targeted feedback from a dedicated group of coordinated investors are encouraged to apply to participate in interactive pitching, partnering, and one-to-one meetings at RESI Europe.
As is customary at the turn of the year, we have taken the opportunity to take a look back at financing deals we covered since issue#1, which went live in April last year. Together, these data offer a snapshot of how capital flowed into early-stage, preclinical therapeutic startups in 2025 — and where it did not.
Before diving into the numbers, it is worth qualifying that this analysis captures only publicly disclosed financing rounds, rather than the full universe of early-stage biotech funding. An increasing fraction of preclinical companies now operate in stealth, in part because of fast-moving competition from regions such as China. As a result, the figures presented here likely undercount the true level of early-stage activity.
From the start of our coverage in Q2 2025 through the end of December, we reported 195 preclinical financing rounds. Because Haystack Science focuses on discovery-stage and pre-IND companies, this number excludes financings for assets already in clinical development. Even so, the dataset provides a useful lens on early-stage investor behavior.
Independent industry analyses paint a consistent picture. Multiple sources indicate that 2025 was a year in which venture capital shifted toward later-stage, clinical-stage deals, which were fewer in number but larger in size. This trend was reinforced by ‘Q4 2025 Biopharma Licensing and Venture Report’, presented at the JP Morgan conference. According to JP Morgan, 2025 saw just 191 seed and Series A financings, the lowest total since 2020.
Seed and Series A investment slowed through Q4 2025, losing momentum built earlier in the year and further lagging Series B and later rounds. The slowdown in early-stage funding reflects heightened diligence standards and longer decision timelines for early-stage startups. Source: JP Morgan
According to the Haystack Science data sample, no venture fund made a series A investment in more than three companies last year (these series A financings ranged from $8–300 million, with a median of $42.5 million). As the deals that Haystack tracks are only the publicly disclosed subset, we expect our sample is skewed to companies that raised larger sums. In the deals we tracked, the most bloated series A ($300 million) went to Cambridge, Mass.-based Lila Sciences, a generative ML model powered startup building “autonomous, closed-loop experimentation using generative ML models to generate drug mechanism hypotheses, test them robotically in the lab with minimal human intervention, and iteratively learn from results.” Lila was backed by megafund Flagship Pioneering and General Catalyst.
21 funds invested in more than one series A round. These were: Arch Ventures, Atlas Venture, Lightstone Ventures, 3E Bioventures, Access Industries/Biotechnology, BGF, BVF Partners, Canaan Partners, Cormorant Asset Management, Dementia Discovery Fund, Eight Roads, Johnson & Johnson Innovation – JJDC, Khosla, Omega Funds, Orbimed, Polaris Partners, Samsara, Santé Ventures, Sofinnova Partners, The Column Group, and Versant Ventures. No fund invested in more than 3 series A investments in last year’s sample.
Further back in the pipeline, we tracked 60 deals. These seed financings—which ranged from $1.1–54.5 million with a median of $10.45 million—were mostly for smaller amounts ($1–$30 million), with a few much larger financing amounts. Overall, 85 different funds, family offices, angels and individuals participated in funding preclinical therapeutic startups in 2025. Of these 85 sources of financing, only 7 financed more than one company. The takeaway from this is that most (>90%) of companies at the seed stage receive funds from a completely unique set of investors.
The 7 financing entities involved in more than one seed deal were: AdBio Partners, Kurma Partners, NRW Bank, Ackermans & van Haaren (AvH), Bioinnovation Institute (BII), ClavystBio and ExSight Ventures. It is noteworthy that two of these funds are based in Paris, France: AdBio Partners and Kurma Partners. AdBio specializes in early-stage investments across Europe with a ~€86 million ($102 million) fund raised in 2021 focusing on oncology, immunology, and rare diseases. Kurma is part of the Eurazeo group, managing >€600 million in assets across several funds focused on early-stage therapeutics and diagnostics.
NRW.BANK, based in North Rhine-Westphalia, Germany, invests in innovative biotech companies focusing on tech-driven healthcare, bio-digital integration, and novel platforms for data/discovery, aligning with broader innovation goals. They appear to be an important source for the small scattering of financing (13) deals in German-speaking countries. NRW works closely with AvH, an Antwerp, Belgium-based diversified holding company and investment firm, with AvH Growth Capital a proactive investor in early-stage companies like DISCO Pharmaceuticals and Evla Bio.
Another very interesting seed funder is BII in Copenhagen Denmark. The institute provides in-kind grants of up to €3 million for bridging translational studies in European academic institutions. For those projects that progress to a company build, a combination of convertible loans of €500K (Venture Lab) and then €1.3 million (Venture House) are made available to complete seed funding. As of January 2026, BII has supported over 130 early-stage life science and deep tech companies, with many attracting significant external funding. This month, there was news that Novo Nordisk has just plowed another $856 million of funding into BII.
The United States continues to dwarf other countries in its ability to galvanize preclinical biotech startups receiving seed or series A financing, with the UK a far second place (itself hosting double the number of startups of other non-US countries). Source: Haystack Science
Overall, in terms of the location of where most investment is occurring, our analysis reveals the capacity to host startups is expanding across the globe, with at least 19 countries hosting one preclinical startup that received funding in 2025. These countries were: USA, UK, France, Switzerland, China, The Netherlands, Canada, Denmark, Germany, Belgium, Japan, Spain, Israel, Australia, Ireland, Norway, Portugal, South Korea and Singapore. Perhaps the prominence of France as a location for preclinical therapeutic startups was most surprising from our sample. Interestingly, a lot of ex-US startups now also have a US (usually Cambridge, Mass.-based) headquarters. Digging deeper, 85 different cities around the world host a startup that obtained financing (pre-seed to series B) in 2025, with 20 cities hosting two or more. As expected, the Boston cluster led with 28 preclinical therapeutic startups, the Bay area hosted 19, and the UK’s Golden Triangle had 13. Of the following pack, some interesting standout cities were Paris, France (with 5 in our sample) and New York City (with 7), the latter long in the shadow of its Boston neighbor.
Cities hosting two or more startups that received seed or series A funding in 2025 (85 different cities hosted at least one biotech startup receiving financing). Boston and the Bay Area in the United States and the UK’s Golden Triangle (London, Cambridge and Oxford) are the most successful biotech clusters, far ahead of rest of the world. Source: Haystack Science
In terms of the disease areas attracting early-stage investor money, cancer dominates, comprising the focus for 34.4% of the funding raises. This is slightly lower than the biopharma sector as a whole, where cancer comprises up to 45% of pipelines. Following cancer, neurodegenerative disease, autoimmune disease and inflammatory disease all figured prominently. The uptick in deals for companies tackling CNS disorders has been a rolling theme recently, given the burden of neurodegenerative disease and dementia on public health systems and the paucity of disease-modifying treatments. With the continuing stampede around GLP-1s/incretins, there was also a healthy number of metabolic/ endocrine disease startups financed.
Disease focus of pre-seed, seed, series A and series B deals for preclinical startups tracked by Haystack Science in 2025. Source: Haystack Science
One last area we looked at was the type of therapeutic being financed by investment groups. Here again, the pharmaceutical industry’s traditional workhorse, the small molecule, remained pre-eminent in 2025, comprising 24% of financing deals in pre-seed, seed, series A and series B financings that were in the preclinical stage. Established modalities like monoclonal antibodies (mAbs) were a common focus. And there was a resurgence of interest in recombinant proteins and peptides (likely boosted by the focus on incretins and the metabolic disease and obesity space). Of new modalities, antibody-drug conjugates, bispecific and multispecific antibodies, antisense oligonucleotides (ASOs), small-interfering RNAs (siRNAs) and chimeric antigen receptor (CAR) immune cell (T cell and NK cells) also were to the fore, each making up around 6% of all the early-stage deals we tracked. A type of therapeutic gathering increasing attention is clearly the induced-proximity therapeutic sector (including the different flavors of PROTACs, DUBTACs and molecular glues). Finally, although a great deal has been mentioned about investor apathy for gene editing and gene therapy, these also captured 3-4% of the deals.
Type of therapeutic modality in preclinical startups receiving pre-seed, seed, series A and series B funding in 2025 that were tracked by Haystack Science. Source: Haystack Science
Myonerv is developing a new approach to stroke rehabilitation that aims to expand access to intensive, effective therapy beyond the clinic. Following their recent win at the Innovator’s Pitch Challenge at RESI London, the team is advancing a wearable neurotechnology designed to help patients regain upper-limb movement through intention-driven stimulation and remote clinical support. We spoke with Myonerv to learn more about the problem they are addressing, their technology, and what comes next as they move toward clinical trials and global partnerships.
Caitlin Dolegowski (CD): For readers who may be new to Myonerv, how do you describe the company’s mission and core technology?
Sam Kamali (SK): Myonerv is a breakthrough British neurotechnology solution designed to transform stroke rehabilitation through an active, remote-operated wearable medical device that restores movement in patients with post-stroke upper-limb paralysis (paresis). A wearable, non-invasive neurostimulator that helps retrain movement after stroke by detecting a patient’s intention to move and delivering targeted electrical stimulation to augment that movement. This “closed-loop” approach is supported by scientific evidence showing that synchronising stimulation with a person’s voluntary effort can enhance neuroplasticity – the brain’s ability to rewire and relearn lost movements.
Unlike traditional electrical stimulators, Myonerv uses flexible bioelectronic materials to create soft, reusable electrodes that conform comfortably to the arm. The system is designed to be lightweight, easy to apply, and suitable for both clinical and home environments. It will also allow therapists to monitor progress and support patients remotely, helping expand rehabilitation capacity without increasing staff burden.
CD: What problem are you addressing, and why is now the right time for your approach?
SK: Partial paralysis after a Stroke affects 70% of all survivors, approximately 900,000 new patients annually in the UK, DACH and USA. Despite clear evidence that intensive, early, and consistent rehabilitation improves outcomes, most patients in the UK receive only 45 minutes of therapy per day in hospital and as little as 1 hour per week after discharge – far below nationally recommended 3 hours per day. We believe the resulting “plateau” in stroke recovery is not biological, but due to the lack of therapy access and intensity.
Myonerv directly addresses this challenge by developing a first-in-class wearable, closed-loop neurostimulator that enables continuous, intention-driven rehabilitation both in-clinic and at home. It combines bioelectronic sensing, software-assisted control loops, reusable polymer electrodes and remote therapist connectivity (capabilities not currently available in NHS or international markets). The innovation advances beyond the state of the art in its miniaturisation, accuracy, sustainability, and ability to extend clinical rehabilitation into the community setting.
CD: What stood out most to you about competing in—and winning—the Innovator’s Pitch Challenge at RESI London?
SK: “It’s so refreshing to hear such a good pitch, after such a long time”. These were the words that stuck with us from our judge, Soyoung Park, General Partner at 1004 Ventures.
This echoed the depth of excitement from investors throughout the event. The judges and audience deeply understood both the clinical problem and the commercial challenge of scaling medical technologies within healthcare systems. People were enthusiastic about the prospect of a remote-controlled rehabilitation device that can exponentially increase the amount of therapy received by patients. Winning wasn’t just validation of the technology - it was validation of the need and our ability to change the state of healthcare worldwide.
CD: How has the exposure from RESI London impacted conversations with investors or strategic partners so far?
SK: RESI London has materially accelerated conversations. Since the event, we’ve seen increased inbound interest from international investors and strategic partners across Europe and the US, particularly those focused on neurotechnology, digital health, and rehabilitation.
The win has acted as a strong credibility signal – shortening diligence cycles and shifting discussions toward clinical milestones, regulatory strategy, and partnership structures rather than basic validation. It has also opened doors to potential manufacturing and healthcare delivery partners who see Myonerv as an international platform, not just a single product.
CD: Where is Myonerv currently in terms of fundraising or partnership strategy?
SK: We have officially opened a £2 million pre-seed priced round. Our strategy is to combine the £215k in non-dilutive funding with targeted private investment to de-risk the technology before scaling. What is promising is that we are receiving non-dilutive funding faster than we can announce it, with a recent admission into the Founders Factory x Innovate UK (Hospital to Home) Biomedical Catalyst Accelerator, which enables a£100k grant for us to perform our first in-patient trials.
This touches on our recent partnership arrangements, as we are working closely with several patient networks and the Cambridgeshire & Peterborough Foundation Trust (CPFT), an NHS Trust overseeing hospital networks across the East of England. CPFT has agreed to sponsor our clinical trials, which fast-tracks the process for trial approvals, recruitment and secure documentation of data. CPFT has shown great enthusiasm in being our first pilot sites for Myonerv. This is only a small part of our partnership arrangements, as we have several more with other hospitals across the UK who have given us Letters of Interest to give Myonerv to 1,200 patients per year once the device is available in the market.
We are now looking to build on the network we have built in Cambridge thanks to the £120k grant won from ARIA as part of Cambridge NeuroWorks, tasked with developing a first-in-class scalable neural interface for the world. Our next step is to visit RESI JPM in San Francisco on January 12th 2026 to create more partnerships with the US network as we look to expand our reach globally.
CD: What milestones are you most focused on over the next 12–18 months?
SK: Our primary focus is delivering on the tech. We are currently finalizing our functional prototype with the Manufacturing Technology Centre, a major UK-based factory, to test on participants with approvals from the University of Cambridge. Our shiny new prototype will be ready in time for an exciting Myonerv demo at RESI Europe in Lisbon, on 23rd March 2026!
We are then looking to build on this to develop a TRL6/7 alpha device and completing a feasibility clinical study in stroke survivors. In parallel, we are advancing our regulatory pathway, quality management systems, and health-economic evidence to support adoption by healthcare providers. Having secured regulatory and commercial partners who will help navigate our pathway through into international markets.
We are simultaneously focused on strengthening manufacturing readiness, expanding our clinical and patient engagement network, and closing our pre-seed round. Together, these milestones position Myonerv for scale - clinically, commercially, and globally.
Caitlin Dolegowski (CD): Could you introduce TrilliumBiO and share your core focus areas in life sciences?
Laura Vivian (LV): TrilliumBiO is a biomarker discovery company specializing in the development and commercialization of novel diagnostic tests to translate scientific discoveries into real-world clinical impact.
The company has launched over 100 assays, collaborates with partners domestically and internationally, and processes over 500,000 samples annually through a multi-accredited, CLIA-certified laboratory. We work with industry innovators in biotech and pharma, as well as academic medical centers, foundations, and patient advocacy groups. Headquartered in Maryland, just outside Washington, D.C., we operate within the nation’s third-largest biopharma hub. Our multidisciplinary leadership team brings decades of experience delivering value to patients and partners.
Our core focus is expanding access to critical areas of testing that align with emerging therapeutics and scaling diagnostic solutions that support the development and adoption of new treatments.
CD: What types of early-stage companies or technologies are you most interested in meeting at RESI?
LV: First, we are excited to be able to sponsor RESI JPM 2026 and be part of this great community. Thank you for having us.
We believe we are ideally positioned at the intersection of the life sciences ecosystem to create enormous value for our partners. We’re especially interested in engaging with companies advancing novel therapeutics and diagnostics, investors seeking biomarker and clinical diagnostic expertise for their portfolio companies, and organizations with technologies to in-license or co-develop. Our team brings speed, efficiency, and deep expertise in biomarker strategy and development to help accelerate that journey.
CD: What are some of the key scientific or commercial challenges your team is focusing on solving in the coming year?
LV: At TrilliumBiO, we see ourselves as partners from discovery through delivery, working alongside our clients across R&D, regulatory milestones, and clinical use. That partnership means solving critical barriers that often slow diagnostic development, limit patient access, and delay therapeutic approvals.
We’re not only able to bring new assays to market; we also scale testing volume and accelerate the commercialization of existing assays. Our regulatory expertise and audit readiness gives partners confidence that FDA submissions will succeed, ensuring progress isn’t stalled by compliance hurdles.
Education is foundational to our work, strengthening disease awareness among both patients and providers. With the support of more than 15,000 in our physician network, we make sure that every test result is clinically meaningful and actionable.
CD: Is there anything you’d like the RESI community to know about TrilliumBiO’s mission or upcoming milestones?
LV: We recently announced a strategic partnership with Oncobit, an international leader in precision oncology, to bring advanced monitoring solutions for uveal melanoma, including molecular residual disease (MRD) testing, to the U.S. We’re also preparing awareness initiatives around rare diseases like lymphangioleiomyomatosis (LAM), supported by our VEGF-D assay, and blood-based biomarkers that enable earlier detection of Alzheimer’s disease. Our mission is to advance diagnostics that make a meaningful difference in patient care.
The RESI community should stay tuned, as we’ll be sharing more about these milestones and others soon.
CD: Are there any recent accomplishments that you want us to highlight? (Awards, Grants, FDA Approvals, Social Corporate Responsibility programs, etc.)
LV: We were honored to be named a finalist for the Emerging Life Sciences Company of the Year at the 2025 ICON Awards presented by the Maryland Tech Council, recognizing innovation and impact in the state of Maryland’s life sciences sector. Building on that momentum, we secured FDA approval for a rare disease direct to consumer test within just six months, a milestone that reflects our ability to rapidly translate discovery into patient-ready diagnostics. Alongside these achievements, we continue to strengthen partnerships with patient advocacy groups, ensuring that our breakthroughs are paired with meaningful support for the communities we serve.
On December 9, the Italian charity Fondazione Telethon made waves by becoming the first non-profit organization to obtain FDA approval for an advanced therapy: Waskyra (etuvetidigene autotemcel) is an ex vivo lentiviral gene therapy indicated for the rare immune deficiency Wiskott-Aldrich Syndrome. Fondazione Telethon’s accomplishment underscores the impact that philanthropic organizations can have on drug discovery and has rightly been celebrated by patient-advocacy groups working to develop therapies for other conditions of limited commercial interest. How can this wider universe of disease foundations emulate Fondazione Telethon’s achievement and leverage the lessons from Waskyra’s approval?
Drug development for rare and ultra-rare conditions faces multiple challenges: limited understanding of the disease, paltry funding, a lack of business models providing a return on investment, regulatory obstacles, manufacturing and distribution barriers, and so on. For all these reasons, venture capitalists and pharma companies have shied away from diseases that, like Wiskott-Aldrich Syndrome, affect small populations of patients. This is the unspoken dirty secret of modern medicine. Current commercial drug development is unfit for >90% of all known diseases.
According to an analysis of the Orphanet database, >80% of rare diseases affect <1 patients per million people (~0.5% of the total number of patients living with a rare disease). By contrast, ~4% of rare diseases affect 100–500 patients per million people (>80% of the total number of patients), enough to attract commercial interest. Source: Drug Discovery Today
With the biopharma industry steering clear of these conditions, patient advocacy groups and other charities are trying to fill the void. According to a recent study commissioned by the US Department of Health and Human Services (HHS), 585 advocacy groups fund “medical product development” activities in the United States. Why has it taken an Italian non-profit organization to be the first to cross the US FDA approval finish line?
The organization responsible for development of Waskyra is the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), a 30-year-old partnership between Telethon Foundation and Milan’s Ospedale San Raffaele. Over those three decades, SR-TIGET has raised over half a billion euros in philanthropic capital to build internal capabilities equivalent to those available in a clinical-stage biotech company: target discovery, preclinical modelling, regulatory strategy, phase 1/2 clinical trials and registration. In other words, unlike most patient foundations and groups, this organization has accumulated the resources to generate the data necessary to walk the full path to approval, independently of the need to collaborate with a pharmaceutical company.
Out of the 585 patient advocacy groups cited in the HHS report, only 11 operate with their own research staff and lab space. In contrast, 106 advocacy groups fund life-science companies, and 536 fund academic or medical institutions. This implies that, at most, only 1.9% of US patient groups use a model that shares at least some similarities with SR-TIGET’s. This is important to emphasize because having all these in-house capabilities makes an organization less dependent on industry partnerships, which can be difficult to secure in the first place and are subject to change if economic conditions and/or company priorities alter.
Patient advocacy organizations (PAOs) use multiple types of arrangement to advance their missions, but only a small percentage conducts in-house research and/or clinical trials. As the categories are not mutually exclusive, individual advocacy groups may be included in multiple categories. Source: Lee, KM et al., May 2025
Of course, it would be disingenuous to expect all patient foundations to adopt the SR-TIGET model. According to the HHS report, the mean annual revenue of an advocacy group capable of funding clinical trials is ~$32 million, with their median annual revenue at ~$3.5 million. Most of the 585 charities have no hope of achieving these financing levels, particularly those advocating for patients living with ultra-rare conditions. At the same time, these figures represent the reality of commercial development, and they should be part of the calculus used by patient advocacy groups to define the scope of their activities and inform their fundraising strategy.
Mean (blue) and median (red) revenue of patient advocacy groups that fund medical research. As the categories are not mutually exclusive, individual organizations may be included in multiple categories. Source: Lee, KM et al., May 2025
It is worthwhile noting that Waskyra is not Fondazione Telethon’s first rodeo. SR-TIGET was responsible for much of the work behind two other approved ex vivo lentiviral gene therapies for ultrarare conditions: Strimvelis (for ADA-SCID; European approval in 2016) and Lenmeldy (for metachromatic leukodystrophy; European approval in 2020; FDA in 2024). In both cases, the organization partnered with for-profit companies to take the drugs to market, providing SR-TIGET with crucial training in the drug-approval process before they achieved their recent independent success with Waskyra. At the same time, those early experiences made it painfully clear that the story does not end with regulatory approval, as many without experience of developing medicines assume.
In 2018, Strimvelis, which had been developed by SR-TIGET in collaboration with GlaxoSmithKline, was acquired by Orchard Therapeutics along with the rest of the pharma’s rare disease gene-therapy portfolio. After taking the therapy to approval, however, Orchard pulled the plug and decided to cease marketing of the therapy. Fondazione Telethon then stepped in and had to arrange the transfer of the marketing authorization from the company to the foundation. Although SR-TIGET has been able to make the therapy available in Italy, Strimvelis remains unavailable elsewhere in Europe. This is unsurprising as setting up distribution networks across continents requires deep expertise and investment, and has long been the sole purview of commercial organizations.
In the case of Waskyra, the manufacturing and distribution strategy for the United States is not yet clear, but a week after the FDA decision, Fondazione Telethon signed a memorandum of understanding with the Orphan Therapeutics Accelerator (OTXL) under which Orphan Therapies (an OTXL subsidiary) will become the exclusive commercialization partner for the therapy. OTXL is a separate, US-based, non-profit organization focused on the clinical development of “shelved” ultra-rare disease treatments. That two independent non-profit organizations have come together to deliver a life-changing therapy to patients is of great significance and perhaps underappreciated by the wider community. It will be interesting to see how this partnership evolves, particularly with regards to pricing.
Indeed, pricing has been another thorny issue for Fondazione Telethon. The cost of Strimvelis is reportedly ~€600K. Between July 2023 (when the foundation obtained the marketing authorization) and the end of 2024, SR-TIGET has treated only two ADA-SCID patients (~14 children are born every year with the disease in Europe). Of most concern, the associated costs for these two treatments were €4.7 million. Although Fondazione Telethon is a non-profit entity, multi-million Euro losses of this kind simply are unsustainable. It will therefore be important that the foundation sets a price of Waskyra on the US market where it can at least recoup the costs of its treatment — if not make a return that it can invest back in further R&D efforts.
Which brings us to perhaps the most important takeaway from SR-TIGET’s Waskyra approval. It is striking how this foundation has focused very heavily on the development of gene therapies, and in particular ex vivo lentiviral gene therapies. Luigi Naldini, leader of SR-TIGIT, is a pioneer in the study of lentiviral vectors, and a lot of the research conducted at the institute over the years has focused on the optimization of vectors and on understanding the biology of hematopoietic stem cells with the eventual goal of fixing disease-causing mutations. According to the SR-TIGET website, the organization has treated ~25% of patients who have received hematopoietic stem cell-based gene therapy worldwide.
In contrast, most patient groups have a starting point around a specific disease (or a subset of related diseases) for which drug-discovery projects are launched, often using multiple therapeutic modalities to have as many “shots on goal” as possible. These are two fundamentally different approaches. SR-TIGET has focused on one therapeutic modality and then deployed it across different diseases; most other foundations focus on one disease and then invest in many different therapeutic modalities.
Ultra-rare drug developers and patient groups should take note: an increasing body of data suggests that organizations achieving development success have adopted a similar platform-based approach to bringing therapeutics to patients. And the reason for this is simple: putting together an entire discovery, commercialization and distribution apparatus for more than one therapeutic modality is simply unaffordable for most independently funded non-profits.
Elsewhere, one might argue that, in base editing, we are also starting to see yet another example of a modality hub emerge. Following the success of base editing around CSP1 for baby KJ (highlighted in Issue #6 of The Needle), the Center for Pediatric CRISPR Cures is building a hub around gene editing R&D expertise — an initiative that the Innovative Genomics Institute’s Fyodor Urnov is also promoting.
What does all this mean? We would suggest that academic medical centers and patient foundations interested in developing ultrarare therapies should consider the platform-based approach as an efficient way to deploy their capital. Evidence is clearly building that focusing on one modality works. For therapies beyond that single modality, organizations might be better served by identifying another resource-rich ‘hub’ organization for development programs in their disease.
Another advantage of a large platform-based hub approach with a host of different disease spokes is that it would result in a diversified portfolio of projects in which each project is a separate shot on goal. This may achieve the scale to deliver a successful drug and, therefore, generate income. In fact, MIT economist Andrew Lo has used financial-engineering techniques to show that a portfolio of ultra-rare disease projects could generate a return on investment exclusively from the sale of FDA’s Priority Review Vouchers (PRVs), which pharma companies seek to acquire for a median >$100 million. Although the reauthorization of the PRV program by the US Congress is uncertain, we think this is a tantalizing insight because it points to a sustainable path for the development of ultra-rare therapies.
PRVs were created by the FDA to incentivize the development of treatments for rare pediatric diseases, allowing companies to get a faster regulatory review for any future drug. Crucially, PRVs can be sold to other firms. The graph shows the known prices that pharma companies have paid to acquire a PRV since they were launched. Source: BioSpace
2025 has been a landmark year for ultrarare therapies. Besides the FDA approval of Waskyra, the successful use of base editing to treat CPS1 deficiency in Baby KJ in just seven months, the acceptance of >160 patients into n-Lorem programs, and the administration of several gene therapies to ultrarare patients (Urbagen, an AAV-9 gene therapy for CTNNB1 syndrome being yet another recent example) suggest that ultrarare disease treatments are finally gaining momentum. With SR-TIGET, n-Lorem, Nationwide Children’s and Elpida showing the way, perhaps a development model is finally emerging to treat these debilitating childhood diseases that devastate too many families around the world.
By Dennis Ford, Founder & CEO, Life Science Nation (LSN)
Life Science Nation (LSN) presents the RESI JPM 2026 Program Guide for its flagship hybrid conference, held January 12–13 in person at the San Francisco Marriott Marquis, followed by three virtual partnering days on January 14, 19 & 20.
RESI JPM connects early-stage life sciences innovators with global investors during the heart of JPM Healthcare Week. Highlights include the Innovator’s Pitch Challenge, featuring 96 emerging companies presenting to investor judges, along with expert panels, interactive workshops, and a diverse exhibitor showcase spanning biotech, medtech, diagnostics, and digital health.
Positioned at the center of JPM Week, RESI JPM offers unmatched access to capital, partnerships, and industry insight. Registration is now open.
The firm is focused on therapeutics companies and does not invest in medical devices, diagnostics, or digital health. The firm is open to considering assets of very early stages, even those as early as lead optimization phase. The firm considers various modalities, including antibodies, small molecules, and cell therapy. Currently, the firm is not interested in gene therapy. Indication-wise, the firm is most interested in oncology and autoimmune diseases but has recently looked at fibrotic diseases and certain rare diseases as well.
The firm is opportunistic across all subsectors of healthcare. Within MedTech, the firm is most interested in medical devices, artificial intelligence, robotics, and mobile health. The firm is seeking post-prototype innovations that are FDA cleared or are close to receiving clearance. Within therapeutics, the firm is interested in therapeutics for large disease markets such as oncology, neurology, and metabolic diseases. The firm is open to all modalities with a special interest in immunotherapy and cell therapy.
A strategic investment firm of a large global pharmaceutical makes investments ranging from $5 million to $30 million, acting either as a sole investor or within a syndicate. The firm is open to considering therapeutic opportunities globally, but only if the company is pursuing a market opportunity in the USA and is in dialogue with the US FDA.
The firm is currently looking for new investment opportunities in enterprise software, medical devices, and the healthcare IT space. The firm will invest in 510k devices and healthcare IT companies, and it is very opportunistic in terms of indications. In the past, the firm was active in medical device companies developing dental devices, endovascular innovation devices, and women’s health devices.
A venture capital firm founded in 2005 has multiple offices throughout Asia, New York, and San Diego. The firm has closed its fifth fund in 2017 and is currently raising a sixth fund, which the firm is targeting to be the largest fund to date. The firm continues to actively seek investment opportunities across a […]
Paula Cerqueira
Laura Vivian
Next Phase Newsletter 