Rocket Pharma COO On Observing Rare Disease Day, And Why It Deserves US Attention

Feb. 28 is Rare Disease Day. It's meant to observe the more than 400 million people globally — 50% of them children — that are affected by rare, pediatric life-threatening diseases such as leukemia, Cystic Fibrosis and Hemophilia to name a few.

But for Rocket Pharmaceuticals Inc. RCKT, every day is rare disease day. The biotech firm, which went public in October, is among the most prominent players in the space with six genetic therapies in its pipeline.

To learn more about the Cranbury, New Jersey-based company, Benzinga spoke with Rocket Pharma president and COO Kinnari Patel about the firm's ongoing quest to find gene therapy cures.

See Also: Rocket Pharmaceuticals Announces Proposed Public Offering Of $100M Of Shares Of Its Common Stock

BZ: When Rare Disease Day was started in 2008, it was intended for Feb. 29 — a rare day that only occurs on leap years — by the European Organisation for Rare Diseases. Does the day get the attention it deserves in the U.S.?

Patel: While Rare Disease Day has started to get attention in the U.S., it deserves even more attention, as the unmet needs for the global rare disease community are staggering. It may come as a surprise to most people that rare diseases have a greater impact on people and families than all types of cancer and AIDS combined. Recent statistics show that over 30 million Americans and 400 million people globally are affected by rare diseases, with children comprising 50% of that number. Sadly, 9 million children living with a rare disease will pass away before their fifth birthday — every year.

Raising awareness of rare diseases has never been more important. First, it’s an opportunity to acknowledge the millions of brave rare disease patients, families and caregivers who fight rare disease every day and gives us a chance to let them know they are not alone.

Second, we must educate the world about the value of early genetic testing, devising management plans and even potentially participating in trials. Approximately 10,000 distinct rare diseases exist today; 80% are caused by genetic mutations, yet only about 5% have an FDA-approved drug treatment. On average, it takes six to eight years for a patient to receive an accurate diagnosis for a rare disease. Early genetic testing can speed up early diagnosis, which can be critical for the patient and their doctors to determine an optimal disease management plan, which may include participating in a clinical trial.

What's Rocket's mission and how did its Rare Disease Day campaign launch?

Rocket’s mission is to seek gene therapy cures. We are seeking to cure diseases by essentially restoring “good” genes so that the “bad” genes are no longer relevant. With promising recent advancements in gene therapy, it’s crucial to educate people about equal access to early diagnosis and investigational therapies, including numerous gene therapy clinical studies that already exist.

We have been celebrating Rare Disease Day from 2017 onwards to raise awareness, provide valuable information to the patient community and advance science. Rocket’s campaign in the U.S. really took off with our “Light Up for Rare” campaign in 2018, with Rocket working with the iconic Empire State Building to change its colors in recognition of Rare Disease Day. This trend continued through the years. Rocket worked with patient advocacy groups in 2021 around the globe to have more than 120 monuments and landmarks light up; this year included NASDAQ Towers in Time Square, the Empire State Building, Niagara Falls and Rome Colosseum.

During the recent LAD-1 trial, all nine patients had shown a 100% survival rate with zero hospitalizations post-therapy. What do you credit that success to?

We are delighted with the positive top-line data from the Phase 2 pivotal trial of RP-L201 for severe LAD-I. We have made significant progress in advancing a world-class pipeline of AAV cardiovascular and LV hematologic gene therapies that aim to correct the root cause of this devastating genetic disease.

Reaching this promising stage is a direct result of Rocket’s innovative approach to R&D following strong science, designing clinical studies incorporating patient feedback, and collaborating with all stakeholders including patients, treating physicians, gene therapy experts and health authorities.

Most importantly, we are committed to drug development that truly puts patients first. As the communities impacted by any one rare disease tend to be small, Rocket has established strong and vital relationships with patients and patient advocacy organizations, which has led to a significantly more informed approach in advancing treatments and to clinical trial design.

When licensing our LAD-I program, Rocket’s founders traveled the globe to meet with patients and families impacted by the disease to understand their journeys and true unmet needs. Ever since, Rocket has been committed to listening to patients every step of the way.

Core to Rocket’s approach to seeking gene therapy cures is the inclusion of patient perspectives at every single stage of the drug development pathway to maximize the opportunity to address a patient's critical medical needs, improving their lives and yield the greatest results throughout the drug development process. Our success thus far — in our LAD-I program and across our world-class pipeline — inspires us to continue our efforts towards developing innovative and effective therapies for patients with rare diseases.

Next: Where Rocket Pharma Stands With Analysts

Image: Courtesy of Rocket Pharma