Panel discussion at USA–India Chamber of Commerce’s BioPharma & Healthcare Summit in Boston / New India Abroad

Breakthroughs in neuroscience, rare diseases and precision medicine are bringing the biopharma industry closer to long-awaited advances, but challenges in regulation, patient selection and clinical trials continue to slow progress, industry leaders said at a high-level panel.

At the USA–India Chamber of Commerce’s BioPharma & Healthcare Summit in Boston, executives said new tools, better targets and emerging modalities are reshaping how companies approach complex neurological and rare diseases.

Dr. Alfred Sandrock, President and Chief Executive Officer of Voyager Therapeutics, said recent advances are beginning to overcome long-standing barriers in neuroscience. He pointed to progress in delivering therapies across the blood-brain barrier. “The whole concept of using transporters to get these across the blood brain barrier… is really important,” Sandrock said.

He said earlier failures in neurology were driven by poor understanding of disease biology. “We didn’t really know the targets to go after,” he said. That has changed, he added, with “highly validated targets by human genetics, human biology.”

Sandrock said new approaches such as biologics, gene therapy and receptor-mediated transport are opening previously “undruggable” pathways. “These are gonna be treatments that are gonna be transformative,” he said.

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Dr. Priya Singhal, Executive Vice President and Head of Development at Biogen, said the industry is at a turning point but must balance innovation with disciplined investment. “We are able to take informed risk because… the innovations are really amazing,” Singhal said.

She cautioned that progress is uneven across stakeholders. “We still have a lag from the perspective of the regulator… and from the perspective of the neurologist,” she said, adding that neurology has long been seen as “the next oncology” but has yet to deliver comparable outcomes.

Singhal said advances in biomarkers are improving clinical development. She cited neurofilament light chain data, which reduced trial sizes from “more than 1400 subjects… to 55 subjects,” enabling faster and more efficient decision-making.

Dr. Ivana Magovcevic-Liebisch, President and Chief Executive Officer of Draig Therapeutics, said the field is entering a new phase in both neurodegenerative and neuropsychiatric diseases. “We are kind of at the dawn… of a breakthrough in Alzheimer’s space,” she said.

She pointed to neuroinflammation and genetically validated targets such as TREM2 as promising areas. At the same time, she said progress in neuromodulation is enabling safer and more precise therapies. “We seem to be able to now come up with more safe and precise neuromodulators,” she said.

Magovcevic-Liebisch said that balancing neurotransmitter systems is critical. “It’s not GABA or… glutamate. Both are critically important,” she said, adding that earlier drugs failed due to lack of selectivity and safety concerns.

Dr. Steven Paul, Founder and Chair of the Board of Seaport Therapeutics, said neuroscience can be broadly divided into neuropsychiatric and neurological disorders, both of which are seeing progress. “We are making progress… a little differently,” he said.

Paul said recent advances in depression, epilepsy and Alzheimer’s research are encouraging. He noted that new treatments are moving beyond traditional mechanisms. “The first new antipsychotic drug that doesn’t work [through] dopamine receptors” has emerged, he said.

He called Alzheimer’s disease the “biggest public health challenge… of our generation,” citing rising prevalence and economic burden. “Currently there are 7 million people in the US with Alzheimer’s… we’re spending about $500 billion,” Paul said.

He said disease-modifying therapies are beginning to emerge. “The first two disease modifying therapies have been approved,” he said, adding that better treatments could arrive “in the next three to four years.”

Dr. David Meeker, Chairman and Chief Executive Officer of Rhythm Pharmaceuticals, said rare diseases present both scientific and economic challenges, but also significant opportunities. “Rare has always been difficult… mostly because small data sets are hard to work with,” he said.

“The business model is absolutely key,” Meeker said, noting that high-value therapies enabled sustained investment in rare conditions.

Meeker said rare diseases are becoming more precisely defined. “Science is getting better… diseases are being fragmented,” he said, allowing targeted therapies with higher response rates.

However, he said regulatory pathways remain inconsistent. “It’s not like rare diseases are handled by one division,” he said, adding that outcomes often depend on individual reviewers and their understanding of the condition.

Singhal echoed that view. “The FDA intends to get more efficient… but… you still need… clear natural history data,” she said, pointing to continued reliance on traditional trial standards.

Panelists said patient selection is a major challenge, particularly in neuropsychiatric conditions. Paul said clinical trials are complicated by heterogeneity. “A given drug probably only works in a subgroup of patients,” he said.

He said new tools such as AI and biomarkers are improving trial design. “We can predict who those people are gonna be before we enrol them in a trial,” he said, referring to the use of EEG data to identify responders.

Wearables and digital tools are also helping measure outcomes. “You can determine whether a drug might be impacting… symptoms,” Paul said.

Despite challenges, executives said collaboration and innovation across modalities will drive progress. Singhal said smaller biotech firms remain critical. “Collaboration is key… to unlock that,” she said.

Sandrock said future breakthroughs will depend on tackling underlying biology, including neuroinflammation. “We’ve only scratched the surface,” he said.

The discussion highlighted a broader shift in the industry towards precision medicine, targeted therapies and smaller, more efficient trials, particularly in complex neurological and rare diseases.

In recent years, advances in gene therapy, biologics and biomarkers have accelerated drug development in areas once considered intractable. Neurological disorders, including Alzheimer’s and Parkinson’s, are drawing increased investment as ageing populations drive demand for effective treatments.

At the same time, rare diseases—often defined by small patient populations—are gaining attention due to improved diagnostics and targeted therapies, supported by regulatory incentives and evolving business models.

Industry leaders said the convergence of science, technology and investment is creating a window for breakthroughs, even as regulatory and operational challenges persist.

 

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