Why I Founded Gene Therapy Consultancy
Dr. Rahul Kaushik
For over 10 years, I’ve been immersed in the world of AAV and Lentiviral gene therapy, contributing to platform technologies and therapeutic programs across diverse diseases. This field is not just my profession; it's my passion.
I’ve seen its incredible potential up close, celebrated its breakthroughs, and, importantly, I've also seen the downfalls of pioneering companies. After critical analysis of past and current events, clear patterns and critical gaps started to immerge that separates success stories from cautious tales.
A Pattern of Solvable Challenges:
The AAV field, while revolutionary, is still maturing. This became particularly evident around 2021. The gene therapy space was experiencing explosive growth, investment was high, and the air was thick with the promise of transformational therapies. Yet, amidst this excitement, a fundamental issue persisted: a scarcity of seasoned AAV experts who had truly navigated the unique, end-to-end journey of AAV product development.
I saw dedicated researchers deeply focused on discovery, while crucial drug development aspects like CMC (Chemistry, Manufacturing, and Controls) were sometimes approached with strategies borrowed from other biologic fields, like monoclonal antibodies – strategies only partially suited to the unique demands of AAVs. Technology companies were racing to create specialized tools, and regulatory bodies were working diligently to understand the nuances of these novel therapies. The path to clinic, let alone commercialization, was incredibly challenging. The market’s cautious reaction to some early commercialized AAV products, even after regulatory successes – like Bluebird Bio's stock plummeting the day they received FDA approval for an AAV drug, an unprecedented event – sent a wave of caution through the entire field.
Once evaluated over $10 B recently being sold for $29 M, sends a clear message: navigating the AAV gene therapy landscape requires more than just brilliant science.
It demands astute strategic planning, deep market understanding, robust manufacturing and commercialization strategies tailored to gene therapies, and an intimate knowledge of the evolving regulatory and reimbursement environments. Choosing to invest in AAV assets, developing the right technology, and crafting a viable path to patients necessitates expert guidance. This is where specialized knowledge becomes paramount.
The Birth of a Mission: Gene Therapy Consultancy
These experiences solidified my conviction. I realized that for AAV gene therapy to truly fulfill its potential, specialized, current knowledge couldn't remain siloed. It needed to be accessible. This led me to establish Gene Therapy Consultancy with following core missions:
  • To democratize specialized AAV knowledge and empower every innovator, biotech, and foundation to overcome critical developmental barriers.
  • Help Investors to differentiate Hype vs Reality
  • Help Technology developers to develop the right tools
- All this is done by curating critical information and creating a knowledge database specifically for the advancement of AAV based gene therapy and not generic biotech best practices.
Today, the field is advancing with a more measured, steady pace. Yet, the need for focused expertise is even more acute:
  • AAV drug developers – from academic spin-outs to established biotechs – require highly specialized help from real experts who are intimately familiar with particular segments of the AAV value chain and not half baked advice from one consultant or ChatGPT.
  • With investor sentiment more cautious and funding tighter, investors themselves need clear, expert guidance to identify and back the programs with the highest probability of success.
  • Technology companies must connect with the right AAV professionals to ensure they're developing tools and reagents that genuinely address the field's evolving needs.
Our Vision for the Future of AAV Gene Therapy

Widespread Patient Access
Life-changing therapies available to all who need them
Streamlined Manufacturing
Cost-effective, scalable production processes
Standardized Approaches
Established best practices and regulatory pathways
Knowledge Foundation
Democratized expertise across the industry
Despite the hurdles, the promise of AAV gene therapy remains immense. The stumbles and successes are collectively paving the way for a more mature, strategically astute field. I often draw a parallel to the monoclonal antibody field, which took roughly two decades to mature into the powerhouse it is today. AAV gene therapy is on a similar trajectory, and it is absolutely here to stay.
At Gene Therapy Consultancy, our overarching vision is to significantly speed up this maturation process for AAV therapies. I along with the Team of extremely talented and active professionals deeply embedded in the AAV industry – are committed to removing as many roadblocks as possible. Because, ultimately, every challenge we overcome together, every AAV program we help advance, brings us closer to delivering on the profound promise of gene therapy for the patients waiting.
The future looks incredibly bright for gene therapy, but its transformative power will be most profoundly realized when the right disease is chosen, the right strategy is meticulously planned, the right technology is expertly developed and implemented, and decisions are guided by comprehensive information and deep expertise. With these elements in place, AAV gene therapies will continue to move from the realm of the miraculous to the mainstream, offering hope and tangible change for countless patients and families worldwide.
I invite you to connect with us and explore how we can support your journey."
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