By Michelle Berg
My Abeona Therapeutics colleagues, Dr. Tim Miller (CEO) and Andre’a Lucca (VP, Communications), and I were able to gather recently with friends, old and new, to enjoy great food and conversation with Huntington Beach as the backdrop. Our luncheon served as a nice pause for everyone to reflect on what has been accomplished and reinvigorated us to continue the focus on bringing forth our therapies for Sanfilippo Syndrome (MPS IIIA and IIIB), Juvenile Batten Disease and Fanconi Anemia. This time together coincided with the annual Global Genes series of events which are intended to bring rare diseases to center stage.
This was my first time attending and I am still working to take in all I heard and saw. The Patient Advocacy Summit, Corporate Alliance meeting, and the Tribute to the Champions of Hope Gala were not only informative and organized extremely well, but also brought forth a multitude of emotions and thoughts as I become more integrated into the rare disease community. What I walked away with is a greater appreciation for the collective empathy that takes place across the span of diagnoses, ways with which our organization can specifically contribute, and new relationships that will be important in advancing Abeona’s Patient Advocacy program. Throughout all of these events, there was such a vibrancy in the interactions that made one feel, well, quite hopeful and certainly capable; part of something greater.
The two-day Patient Advocacy Summit gathered participants from disease specific patient groups and foundations, as well as those from regulatory, legislative, industry, healthcare, and academic backgrounds. All came together to learn about the different aspects and considerations regarding rare diseases, how to share in the work of advancing knowledge and therapies, and how to ensure that the right groups are at the table contributing effectively and efficiently.
Gayatri Rao, Director, Office of Orphan Products Development for the FDA, spoke on how the patient voice can contribute to the regulatory process and improve the design of clinical trials. Her delivery was compassionate and warm while relaying timelines and expectations that aren’t always what people wish to hear. It was encouraging to know the agency is working to better understand the special considerations and tolerances that those impacted by a rare disease have.
Following the Summit, stand-outs who have been integral in uplifting the rare disease community this year were honored during the Tribute to the Champions of Hope Awards Ceremony. Such a moving time to hear of each Honoree’s story and impact upon others, particularly the younger award recipients.
Steven Aldrian provided a beautiful introduction on how he came to know Dr. Maria Escolar, Director for the Study of Neurodevelopment in Rare Disorders Program, Associate Professor, Pediatrics at the University of Pittsburgh. Dr. Escolar, also an Abeona Therapeutics Scientific Advisory Board Member, was honored for her work in Medical Care and Treatment. She shared how her career path made a sharp turn towards rare diseases in 1999 when a mother implored Dr. Escolar to help her daughter, a baby with Krabbe disease. Since then she has served hundreds of families and helped to build the highly reputable Center for Rare Disease Therapy at the Children’s Hospital of Pittsburgh.
The Corporate Alliance meeting provided updates on all of the different committee activities and allowed for feedback and participation that will further ties between industry and Global Genes. We were also pleased to have Dr. Miller invited to participate in the Medical and Scientific Advisory Board meeting that followed.
Finally, we arrived at the culmination of the weekend’s events, The Tribute to the Champions of Hope Gala, complete with a walk on the blue carpet. Once again the Global Genes team did not disappoint and their preparations resulted in a homegrown, organic atmosphere that further tightened the neighborhood feel that had been developing throughout the previous days. The program was entertaining, emotional, truly inspiring…and perhaps the one who stole the show was 12 year old, Ben Lao. He’s a brilliant and completely engaging math whiz who is taking voice lessons; something he showed off while singing Titanium as a duet with Kris Allen. All were impacted by this young champion and wishing for more time with him.
This memorable time provided some of the very best reasons to gather together: to celebrate, to uplift, and to advocate for the families that Abeona serves and truly, for all impacted by rare diseases. I’m grateful to have been part of these events and am feeling even further prepared and energized to continue in my service. Looking forward to next year’s events…Thanks and congratulations, Global Genes team!