Investment portfolios in family offices are geared toward protecting first, and then growing generational wealth, according to Bibhash Mukhopadhyay, moderator of the panel debate entitled “Doing Well Through Doing Good: Investing in Rare Disease Drugs” at the Family Wealth Report Family Office Investment Forum 2026 in New York. This is typically achieved with a traditionally diversified capital allocation approach where a small fraction of discretionary allocation is toward mission-driven outcomes that the family principals are passionate about in spaces or areas where they want to make a difference.

Historically, these spaces have spanned affordable housing, alternative energy, women empowerment, global health, health equity, racial equity and the like, where the power of a company building through solutions architectures and impact measurement frameworks is well articulated. Also, relatively mature frameworks for evaluating the feasibility of investments already exist. No one has really shone a light in the rare disease space. Historically they have been neglected because they are not considered to be “big enough” by themselves i.e. each of these thirty thousand or so rare diseases afflict a small number of patients, but cumulatively, they impact one in every 10 Americans.

The moderator, Mukhopadhyay, has been working on constructing an investment fund structure and an associated ecosystem of capabilities bespoke to rare diseases drug development. This effort would integrate both philanthropic and for-profit sleeves of capital and construct a portfolio of systematic and consistent returns and measure the impact with a novel scale enabling a double-bottom line approach where return on mission and return on capital are equally pertinent. This was the nucleating theme of the panel discussion.

The participants in the panel debate were carefully curated with representation from a private mission-driven foundation, venture capital investor, an operator in a biotech which underwent M&A to deliver return to its investors, a leader who developed a novel model for rare disease development and a matchmaking marketplace for such treatments and a rare disease policy expert, with a close ear to the ground on how the government thinks about rare diseases.

John Parker, a trustee of the Charles Hood Foundation that supports scientific advancements and innovation in pediatric diseases, as well as founder of Springhood Ventures, described the barriers perceived by the pharmaceutical industry and how a new model for drug development is needed. Being positioned both as an investor as well as affiliated with a private foundation, he shared his perspective between goals advanced by the philanthropic approach versus those advanced by a for-profit intent and how the two can co-exist with a common mission.

Craig Martin, CEO of the Orphan Disease Accelerator, a nonprofit biotech focused on expanding development and commercial access to promising rare disease treatments, highlighted the CGTxchange. This platform will enable a crowdsourced model of rare disease drug development, where a matchmaking process will be performed between capital and projects. The projects seeking financing will be listed for individual investors, who are mission-driven to find a cure, to peruse and provide capital for.

Les Funtleyder, who until recently was the CEO of Applied Therapeutics and previously a fund manager investing in healthcare, opined on how an evergreen fund focused exclusively on rare diseases could be an attractive “financial product” in any asset allocators’ portfolio. He walked the audience through the journey in Applied Therapeutics through the ups and downs of both the drug development process and the financial markets, and how ultimately, he was able to generate a return for his investors through acquisition of the company and fulfil an implicit compact with patients who need the drug that they were developing by appropriately resourcing the effort.

Finally, Jack Kalavritinos, founder and principal, JK Strategies, drawing on his expertise in public affairs and health communications, highlighted how new regulatory pathways are being formed and existing ones reinforced. Despite what is claimed in the popular press about political and policy uncertainty, this “ground reality” in the US federal healthcare organizations , should assuage the concerns of a generalist investor, who at the outset perceives investing in biotech and rare diseases, as risky because of the regulated nature of the industry.

The key takeaways from the discussion went back to underlining rare disease drug development as a space that is ripe for consideration of investment from impact-oriented family offices and UHNWIs. Being able to put a smile on a child or a parents’ face is priceless. And if one can make money while doing it, it brings that added layer of satisfaction. 

See more here for an overview of the Forum from our US Correspondent Charles Paikert.