Here's Why Gene Therapy Stocks Are Soaring Today

https://www.fool.com/investing/2019/02/25/heres-why-gene-therapy-stocks-are-soaring-today.aspx

Roche의 Spark Therapeutics 인수를 위한 5조원의 배팅으로 유전자 치료제 회사들 주가 폭등..

오늘 하루에 Bluebird Bio(시가총액 8.2b, 대략 9조원)  +14.16 % 상승...

유전자 치료제 전문 개발 업체들이 향후 제약 업체에서 큰 관심을 받을 것임..

대한민국 유전자 치료제 전문 업체는 바이로메드 ! 

이 좋은 회사에 주식 투자함으로써.. 우리의 노후 생활을 위한 자금 마련과 운 좋으면 남들보다도 더 일찍 은퇴해서 여유롭게 여행 다닐 수 있습니다..

2015년 Bluebird에 기술 수출한 한국업체는 바로 바이로메드 CAR-T 보유 기술...

(주)바이로메드(대표 김용수)는 미래부 바이오의료기술개발사업을 통해 개발한 키메라 항원 수용체(chimeric antigen receptor T cell, 이하 CAR-T) 기반 면역치료제 관련 기술이 미국 바이오텍인 블루버드바이오사(bluebird bio, Inc.)에 기술이전 됐다고 4일 밝혔다. (http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2119879)

ㅇ ㈜바이로메드는 블루버드 바이오와 2015.12.3(목)에 기술이전 계약을 체결, ㈜바이로메드가 보유한 CAR-T 기술을 블루버드바이오가 독점적으로 사용할 수 있는 권리를 부여했다.

□ 이 계약에 따라 ㈜바이로메드는 블루버드바이오로부터 100만 달러의 계약금을 지급받고, 향후 블루버드 바이오에 의해 진행되는 임상개발 진행상황과 인허가 여부에 따라 마일스톤 기술료(질환별 최대 4,800만 달러, 경상기술료 별도)를 나눠서 지급받게 된다.

ㅇ 특히 이번 계약은 블루버드바이오가 이전 받은 CAR-T 기술로 신규 질환치료제를 개발할 때마다 마일스톤 기술료를 별도 지급하도록 체결되어 이후 블루버드바이오의 제품군 확장에 따라 기술이전 전체 계약규모는 더욱 커질 것으로 전망된다.

또한 이번 성과는 국내산업체가 기초연구를 통해 확보한 기술을 미래부가 후속 지원하여, 전임상연구 결과만으로 해외산업체와 기술이전 계약을 체결한 성과라는 점에서 더욱 의미를 가진다.

□ 이번에 기술이전을 받는 블루버드바이오는 미국 보스턴, 캠프리지에 위치한 유명 바이오텍으로 유전자치료 및 유전자가위 기술을 바탕으로 유전질환 및 면역치료제를 전문적으로 개발하고 있으며 이전 받은 기술과 블루버드바이오의 렌티바이러스 기술 및 생산 능력을 결합, 추가 개발 및 상용화를 진행해 나갈 예정이다.

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What happened

Seemingly every company that mentions the term "gene therapy" or "gene editing" is rising today after Roche announced it would acquire Spark Therapeutics for $4.8 billion. That represents a premium of more than 120% for the gene therapy pioneer, which is bringing a treatment for a rare eye disease to the market. The genetic medicine, branded as Luxturna, will cost $425,000 per eye. It has gotten off to a slow start.

The acquisition of Spark Therapeutics shows there's an appetite among established pharma companies for promising genetic cures and treatments. Or at least that's what investors and Wall Street analysts are hoping. The excitement has sent shares of many related companies higher today.

Crispr Therapeutics (NASDAQ:CRSP) and Editas Medicine (NASDAQ:EDIT), just now entering clinical trials with CRISPR-based gene editing tools, rose as much as 24% and 11.8%, respectively. bluebird bio (NASDAQ:BLUE), Solid Biosciences (NASDAQ:SLDB), Regenxbio(NASDAQ:RGNX), and Audentes Therapeutics (NASDAQ:BOLD) each rose at least 16.5% to start the day. uniQure (NASDAQ:QURE) outpaced all peers, however, rising as much as 42.9%.

IMAGE SOURCE: GETTY IMAGES.

So what

Gene therapy is an awfully broad term. There are various approaches being studied in clinical trials attempting to determine the safest, most effective, and most efficient way to correct or edit specific disease-causing genes. In some studies that's done directly in a human body (in vivo), while in others cells are extracted, engineered in a lab, and then injected back into a patient (ex vivo). Sometimes the gene therapy is powered by a modified virus, while other times the editing is attempted enzymatically. Simply put, no one really knows which approach will be optimal for which indications.

That's why it seems investors are extrapolating the logic behind the Spark Therapeutics acquisition a bit too far. The thinking seems to be that if other biopharmas can develop genetic cures or treatments that help patients manage previously unmanageable diseases, then they, too, could be gobbled up by a deep-pocketed pharma company. That's always a possibility, but many of the gene therapy stocks rising today sport pipelines still in the earliest stages of development.

The primary targets of development-stage gene therapies today tend to be in blood diseases, eye diseases, and rare diseases affecting muscle tissues. For instance, Spark Therapeutics developed Luxturna as an adeno-associated virus (AAV) gene therapy for a rare form of blindness. Here's where each company stands regarding its lead drug candidate and genetic medicine approach:

Company	Market Cap	Lead Drug Candidate, Type	Indication(s) (Type)	Development Stage
Crispr Therapeutics	$2.0 billion	CTX-01, CRISPR/Cas9	Beta thalassemia and sickle cell disease (blood)	Phase 1 (ongoing)
Editas Medicine	$996 million	EDIT-101, CRISPR/Cas9	Leber congenital amaurosis 10 (eye)	Phase 1 (planning)
bluebird bio	$8.2 billion	LentiGlobin, lentiviral gene therapy	Beta thalassemia (blood)	Phase 3 (ongoing)
Solid Biosciences	$340 million	SGT-001, AAV gene therapy	Muscular dystrophy (muscle)	Phase 1/2 (planning)
Audentes Therapeutics	$1.3 billion	AT132, AAV gene therapy	X-linked myotubular myopathy (muscle)	Phase 1/2 (planning)
uniQure	$2.0 billion	AMT-061, AAV gene therapy	Hemophilia B (blood)	Phase 3 (ongoing)
Regenx	$2.0 billion	RGX-314, AAV gene therapy	Wet age-related macular degeneration (eye)	Phase 1/2a (ongoing)

DATA SOURCES: YAHOO! FINANCE, COMPANY WEBSITES.

The success of Spark Therapeutics doesn't necessarily mean AAV-based gene therapies will be the best approach, nor does the nauseating hype surrounding CRISPR gene editing mean it'll be the approach for enzyme-based genetic medicines (scientists are already moving on to other technologies for human therapeutic applications).

Now what

Gene therapy and gene editing hold tremendous promise for treating and even curing diseases. However, the swift pace of innovation in biology-based technologies is both an opportunity for investors and a huge risk. Even if one approach works and earns a product marketing approval, it may only be a few years before an even better approach clears late-stage trials and takes over the market virtually overnight. That would be great news for patients, but it promises to create a perpetual stream of volatility for individual investors. Simply put, none of these stocks really earned the bump received today.