A pharmaceutical company known as a global leader in consumer healthcare operates a significant prescription pharmaceutical business. Its consumer healthcare portfolio includes over 250 products and generates approximately USD 2.4 billion in sales annually. In recent years, the firm launched an internal innovation unit that was established with the mission of developing new businesses through open innovation or by acquiring new technologies. The firm focuses on early-stage innovation, particularly seed and Series A companies, and explores opportunities via direct investment as well as collaborative research and product licensing.
The firm targets transformative concepts in healthcare, such as preventative technologies, non-invasive treatments, and early diagnostic tools. The firm typically invests between USD 300,000 and USD 5 million, though the firm does not lead investment rounds. The firm prefers to invest alongside a lead VC or within a syndicate of investors. The firm’s geographic focus lies predominantly in North America and Europe.
The firm has outlined three priority areas of interest: (1) technologies “beyond the pill,” which includes digital therapeutics, software-based interventions, and medical devices, with a strong emphasis on neurotechnology such as neuromodulation, neurofeedback, and biofeedback; (2) advanced therapies, encompassing novel modalities and emerging therapeutic approaches; and (3) sensing and monitoring technologies, including wearables and vital sign sensing. While the firm does not require board representation, it may seek observer rights when appropriate.
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What do analysts think will be the top 10 drugs in the year 2031 (as searched in GlobalData)?
Top 10 in the US in 2031 (USD Millions)
In the US analyst forecasts for 2031, obesity dominates (with immunology, derm and infection for other TAs). Along with the obesity peptides are 2 small molecules and 2 MAbs. The sales are in the tens of billions.
Top 10 in Europe (USD Millions)
In Europe, along with the obesity drugs and dupixent in derm, we see the oncology ADC Enhertu, the ang2 ophthalmology drug Vabysmo, a CNS CD20, and a GI integrin in the top 10. Sales are in the single digit billions.
Top 10 in Japan (USD Millions)
In Japan, obesity is not visible in the top 10. An anti-infective tops the list, followed by CNS, oncology, GI and including heme disorders. There are companies not in the top 20 for global sales. Most of the top 10 have sales below $1B.
Top 10 in China (USD Millions)
For China, obesity is back in the top 10, but Gardasil, an oncology HPV vaccine tops the list. Local company “fast followers” are apparent and most of the top forecasted drugs are not yet launched (presumably a reflection of the rapidly evolving pharmaceutical environment). To get into the top 10, sales are above $500M.
Conclusion: The marketplace for drugs shows considerable variation in different regions around the world.
Just over a week ago, AbbVie paid $2.1 billion for Capstan Therapeutics’ in vivo anti-CD19 chimeric antigen receptor (CAR)-T cell therapy (CPTX2309) for B cell-mediated autoimmune disorders, which is currently in phase 1 testing. In the past few days, EsoBiotec (acquired by AstraZeneca earlier in the year) also published its first clinical data on a lentiviral-delivered anti-B-cell maturation antigen (BCMA) CAR-T approach (ESO-T01) for multiple myeloma, detailing responses in four patients, two of whom showed complete remission. With a host of other companies working on in vivo delivery into endogenous T cells—including Interius BioTherapeutics, Umoja Biopharma, and Orna Therapeutics, the field of in vivo delivered CAR-T cells appears poised at a tipping point.
Ex vivo generation of CAR-T cells (left) is a complex and lengthy procedure, entailing isolation of T cells from patient blood (1), followed by activation, transduction, and ex vivo expansion for several weeks. After undergoing conditioning treatment (2), patients are infused with a bolus of expanded CAR-T cells (3). In the in vivo approach (right), delivery vector (targeted LNPs or lentiviruses, depicted as red dots) are infused directly into the patient, where they encounter T cells and selectively deliver genetic material encoding the CAR (red). Source: Molecular Therapy.
Since transforming the face of cancer treatment in 2017, autologous CAR-T cell therapy has been dogged by logistical issues that have limited commercial rollout and increased costs—the need for leukapheresis, laborious cell harvesting, heterogeneous cell expansion, lengthy turnaround times, and inconsistency of batches—with access limited to just a few clinical centers. Extensive waiting lists can mean many patients die before even being treated, which has driven the search for ex vivo approaches that shorten manufacturing times using fully closed systems and/or miniaturization. Given these challenges, delivery of a CAR-encoding mRNA to a T cell in vivo could be a game-changing technology: No need for viral vectors; no leukapheresis/chemo; no ex vivo manipulation, no requirement for multiple patient hospital visits; no convoluted training of personnel; and no risk of second primary T-cell cancers due to insertional mutagenesis. This last issue has loomed over the field, with all CAR-T therapies carrying black box warnings, although at the end of June the FDA removed all requirements for Risk Evaluation and Mitigation Strategies (REMS).
Writing in Science, the founding team of Capstan Therapeutics, headed by Carl June and Bruce Levine at the University of Pennsylvania and Haig Aghajanian of Capstan, report proof of concept data that functional CAR T cells with antitumor activity can be produced in animal models without any ex vivo manipulation. A key breakthrough in their effort was the development of lipid nanoparticles (LNPs) specifically designed to target T cells and to overcome the propensity of LNPs to accumulate in the liver. To avoid this problem, the authors screened a set of ionizable lipids to identify L829, a lipid that incorporates a tertiary amine headgroup that reduces non-specific interactions with the hepatic system due to its pH-dependent protonation and neutral charge. Ester cleavage sites in the lipid also promote rapid breakdown in, and clearance from, hepatocytes. A final step was to decorate L829 LNPs with a mAb targeting CD5, a T-cell specific marker. The resulting LNP showed limited liver uptake in rodents and non-human primates compared with control LNPs.
To test the potential of L829-containing LNPs to generate functional CAR-T cells, the team engineered them to incorporate 1) mRNA encoding a CAR that binds CD19 on B cells and 2) an antibody targeting CD8+ T cells. These CD8-L829-CD19 targeted (t)LNPs successfully delivered the mRNA in vitro to CD8+ T cells from healthy subjects and from people with B cell-mediated autoimmune diseases. In vivo, these CAR T cells had anti-tumor activity in a humanized mouse model of B cell acute lymphoblastic leukemia.
The Capstan approach to CAR-T cell therapy: An IV bag, a targeted LNP, and an mRNA encoding the CAR of interest. Source: Science.
In cynomolgus monkeys that received repeated doses of CD8-L829 tLNPs containing anti-CD20 CAR mRNA (instead of anti-CD19, which is not cross-reactive between human and monkey), sustained B-cell depletion was observed that lasted for one month. Importantly, reconstituted B cells were predominantly naïve, implying an immune reset — a key therapeutic goal in autoimmunity.
The Capstan in vivo mRNA-encoded CAR T platform eliminates the need for ex vivo manipulation and lymphodepleting conditioning. It avoids the risks often associated with the use of viral vectors that integrate into the genome. It also is transient, allowing dosages to be optimized and quickly stopped if patients suffer adverse events associated with neurotoxicity or cytokine-release syndrome. It will be interesting to see whether the approach is scalable and whether it can open up conditions where long-term CAR-T cell persistence might not be necessary, such as autoimmune disease.
Going forward, an important question will be to determine the potential immunogenicity of the tLNP formulation (especially as the mRNA treatment may be given multiple times), and whether tLNPs cause elevations of human liver enzymes like alanine transaminase or aspartate aminotransferase. Liver toxicity of a novel liposome formulation already caused a clinical hold for Verve Therapeutics’ base editing therapy last year. Future work will also need to define optimal dosing, durability, and long-term safety of this approach. But the work of June, Aghajanian and their colleagues is a compelling advance promising a new era of widely available adoptive T-cell therapies for B-cell driven hematological cancers and autoimmune conditions. A single dose of any of the seven currently approved commercial ex vivo CAR-T therapies costs ~$500,000. A vial of an in vivo treatment is likely to cost an order of magnitude less.
A US-based venture capital firm with a hands-on investment approach and a strong commitment to supporting underrepresented founders invests at the Seed and Series A stages, typically leading rounds with initial check sizes of up to $2 million in equity. The firm makes approximately 1-3 new investments per year and focuses on companies based in the United States.
Within life sciences, the firm invests in disruptive technologies in the medical device sector and is open to all classes of medical devices.
The firm is dedicated to backing diverse founding teams. Its current portfolio includes a significant proportion of companies led by women and minorities.
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A Europe-based venture capital firm manages a dedicated fund focused on diabetes and related conditions. Its investment sweet spot ranges from Seed to Series A, with typical check sizes between $1–3 million. The firm invests globally and is open to both leading and co-investing. Its team brings strong scientific and industry expertise in the diabetes space and actively supports portfolio companies, including through board and observer roles.
The firm focuses on diabetes and related indications such as comorbidities or conditions where diabetes is a key driver (e.g., kidney disease, wound care, and vision loss). It primarily invests in medical devices and digital health solutions, avoiding traditional biotech therapeutics. For hardware, the firm seeks technologies beyond the prototype stage with early data (lab, clinical, or first-in-human). For software, it prioritizes products showing commercial traction or scalable business models. For B2B products, validation through partnerships with medtech firms or healthcare systems is a strong advantage.
There are no specific requirements regarding a company’s founding team.
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A venture capital firm based in Western Europe invests in healthcare companies with typical check sizes ranging from €1–20 million, usually entering at the second or third round of financing and participating through Series B. The firm focuses on companies based in the U.S. and Europe.
The firm invests in diagnostics, digital health, and medical devices, but does not invest in drugs, biotech, or therapeutics. The firm targets companies in the in-development or clinical stages and is indication-agnostic.
The firm does not have specific requirements for management teams and typically takes a board or observer seat on a case-by-case basis.
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A venture capital firm with offices in the US typically makes equity investments in U.S.-based companies, with portfolio companies located on both coasts and throughout the central part of the country. The firm will consider investing at all stages, with a focus on seed and early-stage investments, including founding companies. Investment size is up to $20 million throughout a portfolio company’s path to liquidity.
The firm is currently looking for new investment opportunities in the life science space, with a specific focus on biopharmaceuticals and therapeutic medical devices. The firm has invested in biopharmaceutical companies focused on developing drugs to treat enteral feeding intolerance and disorders that are driven by CD47-mediated signaling pathways, including leukemia, lymphoma, solid tumors, and pulmonary hypertension. In addition, the firm has invested in a medical device developing a minimally-invasive, catheter-based device to re-create valves in deep leg veins. The firm does not look at diagnostics, though may be interested in technology with real-time feedback for procedures.
The firm focuses on identifying and shaping early-stage life science companies in the series A/B rounds to create significant shareholder value. Because of its extensive operating expertise, the firm is able to help entrepreneurs achieve near-term objectives that position their companies for exit.
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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 […]