From The Editor | March 20, 2023

A Mother Picks A CDMO To Advance Treatment For Her Daughter


By Louis Garguilo, Chief Editor, Outsourced Pharma


Until her daughter was diagnosed with Infantile Neuroaxonal Dystrophy (INAD) in 2016, Leena Panwala had been content with her life as a new mom, and selling real estate as a profession.

Today, she’s co-founder (with her husband) and president of INADcure Foundation, a 501C3 nonprofit established to help find a cure for INAD and other forms of PLA2G6-related neurodegeneration (PLAN).

There are no treatments for this rare, terminal, childhood neurodegenerative disorder, nor was there any organization dedicated to aggressively pursuing novel therapies.

There is one now.

During the past half-decade, while the full-time caregiver to daughter Ariya, Panwala has studied the science of the disease. She learned how to established her foundation, including putting together an impressive Scientific Advisory Board, and hiring an experienced consultant.

INADcure is pursuing the development of a potential gene therapy that is being outsourced to a CDMO in hopes of bringing it to human trials. They have ongoing preclinical animal studies in collaboration with Cathleen Lutz, Vice President, Rare Disease Translational Center, Jackson Laboratories, as well as Kathrin Meyer, from the Center for Gene Therapy, Nationwide Children’s Hospital.

Even with all that’s on Panwala’s plate, she was kind enough to speak to me about her experience working with an external development and manufacturing partner.

Points Of Interest

From her home in Fairfield, New Jersey, Panwala starts our conversation by saying, “My background is not in science,” and then adds, “in fact, I did poorly in science in school.”

She could have fooled me.

By the end of our conversation, she provided insights into our entire industry (and a firm grasp of the science her foundation is pursuing).

What initially drew my attention to Panwala was news that the INADcure foundation had selected Andelyn Biosciences as its development and manufacturing partner.

I’d featured Andelyn in a March 2020 editorial titled “U.S. Manufacturing On The Rise: Add Another CDMO To The Heartland.” It had just been spun out from the Nationwide Children’s Hospital as a CDMO specializing in gene therapy.

What I also learned pursing the INADcure story is the key role that a single consultant – who we’ll meet momentarily – played in the organization, and connecting Panwala to Andelyn. This was another reminder consultants have never played a more vital role in outsourcing. (You can register for our March 22nd Outsourced Phama Live webinar on this topic.)

The Selection

But let’s get back to the CDMO: What was INADcure’s selection process?

“The answer is twofold,” says Panwala. “We hired and then worked closely with a consultant who did extensive shopping – comparison of one manufacturer to another – in terms of the production process and knowhow.”

“And obviously," she adds, "pricing was important. We are a nonprofit funded only by families with children afflicted by this devastating disease.”

INADcure ultimately selected Andelyn. For Panwala, one of the reasons was what she had learned through forming relationships with other foundations and organizations.

“Specifically, one of these foundations that caught my attention had gone with Andelyn. They said the entire process was smooth. They were pleased with the communications and the availability of the staff for small nonprofits like us.”

“Now, if I have a question,” she continues, “I get a response right away. We're not waiting days. That is so important to me and our entire organization. They were approachable, and willing to help steer the development of our program.”

Without a science background, she says, “you are not just knee-deep, you are head-deep in all sorts of verbiage that makes little sense to you. To have that sort of one-on-one availability is important.” 

“I send an email, I get a response, in minutes sometimes. I need that reassurance.”

Her, I reply, and every other drug or therapy sponsor around.

The Consultant

I followed up with “the consultant,” who we now name.

He is Neil Hackett, an expert in the clinical translation, planning and execution of toxicology and manufacturing of AAV vectors, as well as writing pre-IND and IND submissions for the FDA. He spent over 20 years in various positions as a research specialist in genetic medicine at Weill Cornell Medical College, until 2012 when he became a consultant.

“Of course INADcure was an unusual customer for vector production,” Hackett tells me, perhaps understating the difference between his client and the “normal” customers CDMOs might expect. 

“I approached a number of academic and commercial vendors to discuss the AAV production project in detail. But Andelyn has a dedicated team to serve non-profit customers.”

Unfortunately, much of the selection process was undertaken under COVID protocols – so no site visits. Nonetheless, Hackett says echoing Panwala, “I had received multiple recommendations from other knowledgeable sources.”

“I knew Andelyn had been through many successful production campaigns. It can serve small and large clients. If the clinical studies of the foundation succeed, they have the capacity for expanded production to approved drug.”

Panwala informed me that INADcure was able to hire Hackett due in large measure from a grant from the Chan Zuckerberg Initiative.

“CZI supports rare disease organizations like ours through their Rare as One Program. Participation in this program has allowed us to build our infrastructure, strengthen our capacity to serve the INAD community, and drive the research forward.”

The First Wrap

Let’s call them the attributes of our narrative:

  • A courageous mother turned indefatigable leader. The establishment of a foundation, supported by an impressive scientific advisory board..
  • The pursuit of a novel gene-therapy for a rare an incurable childhood disease that no others are targeting.
  • Early funding, that among other things, allows for the “first hire” of a super-knowledgeable consultant.
  • Locating and contracting with the right CDMO for the job.

As we end our conversation, my mind is drawn again to that CDMO. What are the exact services they are providing INADcure? How can they be profitable working with non-traditional (very small, and inexperienced) therapy sponsors?

To find out, I spoke with Wade Macedon, Chief Operating Officer at Andelyn Biosciences. That’s next.

But not before another thank you to Leena Panwala, the newest gene-therapy sponsor in our industry.