Recognizing Rare Disease Day

We are trying something new at OLS this month: A Q&A with an Ohio life sciences leader. It is a feature I hope to incorporate more going forward, and I’d welcome your thoughts and insights about this conversation — and about who I should talk with next.?

I recently sat down with Timothy J. Miller, Ph.D. (he/him) , co-founder, President and CEO of Forge Biologics , a central Ohio-based gene therapy development company. Nationally, our drug development models can present challenges to researchers or small companies with gene therapies for patients who need them, especially patients with rare diseases.

Forge is one of the largest Adeno-Associated Virus (AAV) manufacturers in the world — they have the laboratories, facilities and current goods manufacturing practices (cGMP) expertise to help bridge the gap, bringing gene therapies to patients more quickly and efficiently. I talked with Tim about Forge’s work, a recent breakthrough, and a large investment that will help Forge expand to bring treatments to even more families.

Tell me about Forge Biologics. What does the company do?

Forge is a gene therapy development and development engine, focused on enabling access to life-changing gene therapies by helping to bring them from concept to reality. We partner with innovators in the gene therapy community: scientists, physicians, researchers, biotech/pharma companies, and patient groups. With our expertise in gene therapy manufacturing and therapeutics development, we help to champion teams that are navigating the long road from idea to reality.

Late last year, Forge was acquired by Ajinomoto Bio-Pharma Services . Can you tell us about what that investment will allow Forge to do?

After four years in business and incredible growth into one of the world’s largest AAV manufacturers (including Ohio’s largest Series B financing round in history), Forge is now a member of Ajinomoto’s Bio-Pharma Services . Ajinomoto has a fully integrated contract development manufacturing organization (CDMO) business with global sites and extensive experience in development, cGMP manufacturing, and aseptic fill finish services for small and large molecules. Forge has built a strong reputation in the industry for our comprehensive and innovative AAV gene therapy development and manufacturing services. Our partnership will allow us to grow faster, leverage a decades-long commercial infrastructure, and have the backing of a global business investing in our long-term growth for the benefit of our clients and their patients. Additionally, our acquisition further cements Ohio and Columbus as a critical place for gene and cell therapy globally and allows Forge to continue to accelerate our growth—from our facility to our people to the number of clients we can support. We are committed to continuing operations in Ohio where our 200,000 sq. ft. custom-designed facility remains the foundation of our business.

Your work has an incredible impact on patients with rare diseases, and February 29 is Rare Disease Day. Can you talk a bit about why it can be so challenging to get treatments from the bench to those patients, and about how companies like Forge can help?

Starting with some rare disease stats that always astound me and help frame why we focus on this day at Forge. There are 10,000+ rare diseases impacting 400 million people worldwide; 1 in 2 of the people suffering from a rare disease is a child, and three of those will not live to celebrate their 5th birthday. Fortunately, the field of genetic medicine has made incredible strides and is booming now, but still over 95% of all rare diseases do not have a single FDA-approved drug treatment (Global Genes ). Developing genetic medicines is a challenging endeavor with factors such as the high cost, long development time, extensive regulatory hurdles, and the capacity and expertise needed to produce them. These challenges are even more pronounced for rare genetic diseases with smaller patient populations and fewer researchers focused on developing therapies. At Forge, we set out to solve these challenges by recruiting a team of gene therapy experts, building a state-of-the-art facility dedicated gene therapy, and a bespoke platform approach that helps developers scale their programs efficiently and safely. At Forge, we are also developing our own rare disease therapeutics program and guided by a patients-first approach in all of our endeavors. Rare Disease Day is an important day that we honor with our patient community and the communities of our clients to help bring awareness to the needs that exist and the families living with diseases who still need treatments.

Forge recently announced some promising updates from a clinical trial on Krabbe disease, a rare neurodegenerative disease that is generally diagnosed in infants under the age of 6 months, and you have advocated for Krabbe disease screening to be added to the Recommended Uniform Screening Panel (RUSP) for newborns. Can you tell me more about this milestone and what it could mean for families?

For many genetic diseases like Krabbe, newborn screening (NBS) provides access to the accurate early diagnosis and intervention that is so vital in shortening the time to essential treatment. Many of these diseases progress quickly, and without early detection and intervention, a critical window can be lost for effective treatment. Krabbe disease has not been on the federal RUSP, but after significant efforts by Krabbe patient advocacy groups and families, in January of this year, a federal committee (the Advisory Committee on Heritable Disorders in Newborns and Children – ACHDNC) voted in favor of recommending to the Secretary of Health & Human Services the addition of Krabbe disease to the national RUSP. This crucial step will enact eight new states adding Krabbe to their state panels, in addition to the 11 where screening already occurs. This is significant progress on the path to ensuring that every baby born in the U.S. has the opportunity for early diagnosis and intervention for this devastating disease. At Forge, we have an active clinical trial for our novel gene therapy for this disease. Our Chief Medical Officer, Dr. Maria L Escolar, MD, MS , is the world’s leading expert on Krabbe, having pioneered the current standard of care for patients with Krabbe disease. Forge’s FBX-101 is a gene therapy that complements the current treatment to address the progression of the disease that so often leads to rapid decline and loss of mobility in children with Krabbe. Our clinical trial has shown positive safety and efficacy data in patients with Krabbe treated with FBX-101, all of whom were identified through NBS. Timely identification through NBS is vital for the success of not only our gene therapy but all future treatment options.

Central Ohio has been recognized as a growing hub for cell and gene therapy. Why do you think that is? What is it about this state that makes it a good place for Forge to call home?

When we launched Forge, we deliberately built our facility and established our headquarters in central Ohio because of its rich history in gene and cell therapy. This is largely due to the decades of investment in gene and cell therapy research and development at some of our prominent institutions. Coupled with proximity to major universities able to support the technical recruitment needed for our success and access to key investment from the nation’s largest non-coastal VC firm in Drive Capital , we felt Columbus was the best place to call home. The region boasts other significant factors in addition to a legacy industry, access to talent, and investment—it affords a great quality of life, support from our local government, and a community all aiming to move life sciences and the work that we do forward.

My meetings with patients and their families have been among the most profound experiences of my professional career. Hearing their stories and seeing the way medical breakthroughs have helped them crystallize the importance of this field. It puts faces and names on the work we do every day.

Life sciences practitioners discover and develop therapies that save people’s lives and bring people a better quality of life. Ohio’s growing life science ecosystem will no doubt be the catalyst for even more treatments for rare diseases. That’s at the heart of what we do, and why we do it. It’s what I’ll be thinking about today, and what I hope to keep in mind as we work to advance this industry throughout our state.

Best regards,

Eddie Pauline President & CEO Ohio Life Sciences