When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.
We lasted about a year before my living room couldnt take it anymore, she said.
With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.
Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.
The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.
The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.
Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.
Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.
The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.
Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.
The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.
Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.
Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.
For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.
- Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT - February 17th, 2021
- Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and... - February 17th, 2021
- Avance Biosciences Expanding Houston Campus in Support of Cell and Gene Therapy Drug Development - BioSpace - February 17th, 2021
- Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com - February 17th, 2021
- Gene Therapy GS010 Safe, Well-Tolerated for LHON Patients - MD Magazine - February 17th, 2021
- bluebird bio 'baffled' after NICE rejects beta-thalassaemia gene therapy - - pharmaphorum - February 17th, 2021
- Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of... - February 17th, 2021
- GenSight Biologics' gene therapy proves safe in LHON trial - Clinical Trials Arena - February 17th, 2021
- Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference - GlobeNewswire - February 17th, 2021
- Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive - February 17th, 2021
- Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe... - February 17th, 2021
- CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way - FiercePharma - February 17th, 2021
- Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board - Business Wire - February 17th, 2021
- bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes - February 17th, 2021
- Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -... - February 17th, 2021
- Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -... - February 17th, 2021
- Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace - February 17th, 2021
- Gene Therapy Market to Reflect Impressive Growth Rate During 2020-2027 AveXis, Vineti, uniQure NV, Spark Therapeutics KSU | The Sentinel Newspaper -... - February 17th, 2021
- Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence - BioSpace - February 17th, 2021
- Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases - BioSpace - February 11th, 2021
- Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health... - February 11th, 2021
- The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire - February 11th, 2021
- FLT201 Shows Promise as a Gene Therapy for Gaucher Disease - MD Magazine - February 11th, 2021
- NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bio's Gene Therapy for Life-Limiting Rare Blood Diseas - PharmiWeb.com - February 11th, 2021
- Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of... - February 11th, 2021
- Breakthrough gene therapy helps Utah boy thrive - Yahoo News - February 11th, 2021
- Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance - February 11th, 2021
- Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo... - February 11th, 2021
- Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire - February 11th, 2021
- Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma - February 11th, 2021
- FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare... - February 11th, 2021
- Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key... - February 11th, 2021
- FDA approves third gene therapy for large B-cell lymphoma - European Pharmaceutical Review - February 11th, 2021
- Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire - February 7th, 2021
- FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra - February 7th, 2021
- Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace - February 7th, 2021
- The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -... - February 7th, 2021
- AGTC Executives Awarded First Place in the BioProcess International Reader's Choice Awards, Cell & Gene Therapies Category - GlobeNewswire - February 7th, 2021
- Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace - February 7th, 2021
- Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace - February 7th, 2021
- Welsh, Carson, Anderson & Stowe Commits $250 Million in a Strategic Partnership with Kiniciti, a Newly-Formed Platform Investing in Cell and Gene... - February 7th, 2021
- Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)... - February 7th, 2021
- Abeona Therapeutics Announces Clinical Investigator Webinar to Review ABO-102 and ABO-101 Clinical Data Presented at the 17th Annual WORLDSymposium -... - February 7th, 2021
- Decibel and Catalent Sign Development and Manufacturing Agreement for Dual-Vector Gene Therapy for the Treatment of Congenital Hearing Loss - Yahoo... - February 7th, 2021
- Meeting the commercialization challenge of a surging gene and cell therapy market - FierceBiotech - February 7th, 2021
- Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com - February 7th, 2021
- Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - BioSpace - February 7th, 2021
- Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly - Business Weekly - February 7th, 2021
- Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook - Business Wire - February 7th, 2021
- FDA's New Guidance on Cell and Gene Therapy Recommends Assessment of COVID-19 Transmission Risks - Lexology - February 7th, 2021
- After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News - December 17th, 2020
- Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist - December 17th, 2020
- Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,... - December 17th, 2020
- Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire - December 17th, 2020
- Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma - December 17th, 2020
- Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -... - December 17th, 2020
- Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes - December 17th, 2020
- What is gene therapy? - The Star Online - December 17th, 2020
- They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News - December 17th, 2020
- Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire - December 17th, 2020
- Gene Therapy Injection in One Eye Improves Vision in Both - Technology Networks - December 17th, 2020
- Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace - December 17th, 2020
- Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -... - December 17th, 2020
- Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire - December 17th, 2020
- Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace - December 17th, 2020
- Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas - December 17th, 2020
- Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage - December 17th, 2020
- Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire - December 14th, 2020
- ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech - December 14th, 2020
- Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -... - December 14th, 2020
- Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com - December 14th, 2020
- Gene Therapy, Absolutely and For Real | In the Pipeline - Science Magazine - December 14th, 2020
- Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe... - December 14th, 2020
- Rocket Pharmaceuticals in orbit after gene therapy read-out - - pharmaphorum - December 14th, 2020
- Bluebird trumpets long-term data from beta-thalassaemia gene therapy - - pharmaphorum - December 14th, 2020
- ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B - OncoZine - December 14th, 2020
- Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness - IFLScience - December 14th, 2020
- Innovative payment models to support cell and gene therapies on the rise - MedCity News - December 14th, 2020
- Better education needed to give patients improved understanding of gene therapies, new review highlights - University of Birmingham - December 14th, 2020
- Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 - Forecast to 2030 - GlobeNewswire - December 14th, 2020